A Prospective, Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in Hereditary Tyrosinemia Type 1 (HT-1) Patients in Routine Clinical Care in China

This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.

Start Date01 Sep 2025

Sponsor / Collaborator

Swedish Orphan Biovitrum AB

CTRI/2024/03/063760

/ Not yet recruitingPhase 3IIT

Prospective, Randomised, Double blind, Parallel-group Study to Assess the Safety and efficacy of 2/5mg Once Daily Nitisinone among Indian Patients with Alkaptonuria – A randomised control trial. - Nil

Start Date25 Mar 2024

Sponsor / Collaborator

Indian Council of Medical Research

CTRI/2023/05/052626

/ Not yet recruitingNot ApplicableIIT

Pharmacokinetic and Dynamic Correlation for optimum therapy of NTBC for the Treatment of Indian Patients with Hereditary Tyrosinemia Type 1

Start Date15 May 2023

Sponsor / Collaborator-

100 Clinical Results associated with Nitisinone

100 Translational Medicine associated with Nitisinone

100 Patents (Medical) associated with Nitisinone

448

Literatures (Medical) associated with Nitisinone

01 Sep 2025·JOURNAL OF INHERITED METABOLIC DISEASE

Overview of European Practices for Management of Tyrosinemia Type 1: Towards European Guidelines

Review

Author: van Spronsen, Francjan J. ; Das, Anibh M. ; Kuypers, Allysa M. ; Heiner‐Fokkema, M. Rebecca ; Maiorana, Arianna

ABSTRACT:

The introduction of nitisinone (NTBC) and newborn screening for Tyrosinemia type 1 (TT1) enabled preemptive treatment of patients, thereby significantly improving outcomes by preventing liver, kidney, and neurological issues. Treatment goals have shifted from emergency treatment to long‐term care. To evaluate the risk of developing complications with aging, due to TT1 itself or its treatment, long‐term follow‐up is essential. In 2014, an overview of TT1 management practices in Europe was published. Within the Metabolic European Reference Network's subnetwork on amino‐and‐organic acidurias (MetabERN‐AOA), we considered it important to give an update on current TT1 management practices in Europe. An online survey study was performed among members of the MetabERN‐AOA subnetwork, and participants of a workshop on TT1 at the European Metabolic Group Meeting of Nutricia. Findings were compared to existing data from the aforementioned publication from 2014 and previously published recommendations. Thirty‐two centers (16 European countries) completed the survey. Both consistencies and inconsistencies in TT1 management were seen. Inconsistencies were observed in the frequency and methods of follow‐up, dosing of NTBC, and target ranges of biochemical markers. Compared to 2014, key differences included an increased number of patients detected by newborn screening, lower NTBC dosing, and a shift from interest in mainly hepatic to hepatic and neurocognitive outcomes. These results align with trends seen in TT1 recommendations over the years. In addition to numerous consistencies, many aspects in TT1 management still differ widely across Europe, suggesting the need for uniform guidance in clinical management beyond existing recommendations.

01 Sep 2025·INSECT BIOCHEMISTRY AND MOLECULAR BIOLOGY

Killing of Anopheles stephensi mosquitoes by selective triketone inhibitors of 4-hydroxyphenylpyruvate dioxygenase depends on a high protein meal

Article

Author: Dechering, Koen J ; Hartmann, David ; Valdés, James J ; Sheshachalam, Avinash ; Kučera, Matěj ; Perner, Jan ; Vlot, Marnix ; Palusová, Adéla ; Vos, Martijn W

The malaria vector Anopheles stephensi has expanded from Asia into Eastern Africa, posing a growing global health threat due to its adaptive biology and increasing resistance to conventional control methods. Here, we characterise 4-hydroxyphenylpyruvate dioxygenase (HPPD), a crucial enzyme in the tyrosine degradation pathway, and demonstrate its potential as a novel drug target in An. stephensi. Homology modeling combined with molecular dynamics simulations confirmed that key inhibitor-binding residues are highly conserved across mosquito HPPDs and predicted potent inhibition by triketone-based compounds. Using cell-based assay with codon-optimized recombinant expression in Escherichia coli, we screened several triketone and diketonitrile HPPD inhibitors and identified nitisinone as the most potent inhibitor, displaying nanomolar-range IC₅₀ values. Membrane feeding assays showed that nitisinone's insecticidal activity relies on ingestion of a high-protein meal, with haemoglobin identified as the potent dietary factor driving toxicity. These results highlight HPPD inhibition as a promising blood-meal-dependent vector control strategy specifically targeting haematophagous mosquitoes.

01 Jul 2025·Endocrinology Diabetes and Metabolism Case Reports

Combined alkaptonuria and osteoporosis contributing to chronic back pain

Article

Author: Riegler, Anna ; Anand, Gurpreet

Summary:

Alkaptonuria is a rare autosomal recessive metabolic disorder caused by a deficiency in homogentisate 1,2-dioxygenase (HGD), leading to the accumulation of homogentisic acid (HGA) in connective tissues, cartilage, and bones. This accumulation results in multisystem involvement, including early-onset spondyloarthropathy. We present a 54-year-old female from South Tyrol with chronic back and knee pain, accompanied by typical signs of alkaptonuria: ochronosis and darkening of the urine. Molecular genetic testing confirmed the diagnosis of alkaptonuria and identified a previously unreported mutation. Following treatment with nitisinone, a protein-restricted diet, and therapy for osteoporosis, the patient showed significant improvement in symptoms. This case underscores the need to consider rare metabolic disorders in the differential diagnosis of chronic musculoskeletal pain and highlights the importance of early diagnosis and intervention for effective management.

Learning points:

In cases of early or unexplained degenerative spinal and joint changes in younger individuals, consider secondary metabolic causes.In the presence of the symptom triad – ochronosis, dark urine, and arthropathy – alkaptonuria should be suspected.Alkaptonuria is caused by a rare autosomal recessive defect in homogentisate 1,2-dioxygenase, leading to accumulation of homogentisic acid, which primarily results in the destruction of joints and heart valves.Diagnosis is established through biochemical testing and molecular genetic analysis of the HGD gene.Therapeutic options now include nitisinone as a causal treatment (available since 2020); however, due to often delayed diagnosis, symptomatic management and treatment of sequelae (pain control, joint care, and osteoporosis therapy) continue to play a major role.

News (Medical) associated with Nitisinone

06 Nov 2025

·ir.etonpharma.com

Eton Pharmaceuticals Reports Third Quarter 2025 Financial Results

Q3 2025 product sales of $22.5 million, representing 129% growth over Q3 2024 and 19th straight quarter of sequential product sales growth Q3 2025 basic and fully diluted GAAP EPS of $(0.07), non-GAAP fully diluted EPS of $0.04, and Adjusted EBITDA of $2.9 million ET-600 NDA accepted for review, assigned PDUFA date of February 25, 2026 GALZIN® launch ahead of plan; exceeded previous year-end target of 200 active patients Held positive FDA meeting clarifying pathway to KHINDIVI™ label expansion Submitted proposed clinical study to support INCRELEX® label expansion Generated $12.0 million of cash from operations in the quarter Management to hold conference call today at 4:30pm ET DEER PARK, Ill. , Nov. 06, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today reported financial results for the quarter ended September 30, 2025 . “The third quarter was another stellar period for Eton with product sales growing 129% year over year. The impressive results came from strong contributions across our diversified portfolio – ALKINDI SPRINKLE and Carglumic Acid produced strong year over year growth, and INCRELEX and GALZIN delivered major revenue contributions. Both of these recently acquired brands have been reinvigorated with our substantial new investments, including meaningful increases in patient support and education. Our activities with INCRELEX and GALZIN have resonated with the community, improving patient experience and broadening awareness. Both products continue to track ahead of our original expectations,” said Sean Brynjelsen , CEO of Eton Pharmaceuticals . “In addition to our strong commercial performance, we continued to advance our pipeline programs and received favorable news when our New Drug Application (NDA) for ET-600 was accepted for review by the FDA and assigned a Prescription Drug User Fee Act (PDUFA) date of February 25, 2026 . With ET-600, we are excited about the potential to address a significant unmet need within the pediatric endocrinology community, and we have begun full launch preparation activities, eyeing a potential launch in the first quarter of 2026. During the quarter we also held a meeting with the FDA and believe we have reached alignment on a path to broaden the KHINDIVI label,” continued Brynjelsen. “2025 has been a transformational year for Eton. We look forward to closing it out with strong momentum that can propel us to an even more impressive 2026. In addition to continued growth from key products ALKINDI, INCRELEX, GALZIN, and KHINDIVI, we expect to launch ET-600, progress the label expansion programs for KHINDIVI and INCRELEX, and complete ET-700’s pilot clinical study,” concluded Brynjelsen. Third Quarter and Recent Business Highlights 19th straight quarter of sequential growth in product sales and 129% growth over prior year quarter. Eton reported third quarter 2025 product sales of $22.5 million . Year-over-year revenue growth was driven primarily by continued growth in ALKINDI SPRINKLE and Carglumic Acid and the addition of revenues from INCRELEX and GALZIN. INCRELEX relaunch continues to exceed original expectations. INCRELEX was the largest revenue contributor to Eton’s third quarter results. Since relaunching the product, the Company has made considerable progress raising awareness and expanding access to this critical treatment. Eton continues to see an attractive long-term opportunity to invest in education and awareness surrounding severe primary IGF-1 deficiency (SPIGFD), which the Company and key thought leaders believe to be underdiagnosed and undertreated. Eton is committed to expanding INCRELEX access to patients and is pursuing the harmonization of the U.S. and European Union (EU) definitions of SPIGFD as the EU definition encompasses a significantly larger patient population. The Company has submitted a meeting request to the FDA with a proposed study design to support the expanded definition and expects to receive FDA feedback in December. If favorable, Eton could initiate a study in the first half of 2026. The U.S. patient population could increase from approximately 200 to an estimated 1,000 if the EU definition is adopted in the U.S. GALZIN relaunch seeing strong adoption and enthusiastic reception from patient and clinician community. After acquiring the product in December 2024 , the Company relaunched GALZIN in March 2025 . Initial patient uptake has been strong and accelerated in the third quarter as Eton’s rare disease specialists and commercial team further established and strengthened relationships with leading prescribers in the Wilson disease community. The Company recently attended and had a significant presence at the Wilson Disease Association Annual Summit as a top sponsor, where it engaged with patients and industry leaders. Eton’s entry into Wilson disease has been met with enthusiasm from patients, caregivers, and healthcare practitioners, with particular recognition for the Company’s enhanced patient support services and educational efforts. ET-700 pilot study scheduled to initiate in the first quarter of 2026. The Company is scheduled to begin its proof-of-concept positron emission tomography (PET) study in the first quarter of 2026 and anticipates having results in mid-2026. The study is expected to validate the efficacy of the Company’s propriety, patent-pending extended-release formulation, and if positive, would support the initiation of a dose ranging and pivotal clinical trial in late 2026 or early 2027. ALKINDI SPRINKLE continues to post strong sequential and year-over-year growth. ALKINDI SPRINKLE delivered strong sequential and year-over-year growth in both revenue and patients on treatment, boosted by strong output from the Company’s sales force that is now entirely dedicated to pediatric endocrinology. Eton and ALKINDI had a presence at key endocrinology events in the third quarter, including ENDO, the Endocrine Society Annual Meeting. In the fourth quarter, the Company will be presenting or participating in the CARES Foundation Annual Conference and Patient Advisory Summit, SCOPE ( Southern California Organization of Pediatric Endocrinologists ), SPES ( Southern Pediatric Endocrinology Society ), AAP ( American Academy of Pediatrics ) and ILACE ( Illinois Association of Clinical Endocrinology ). Successful FDA meeting regarding development of revised KHINDIVI formulation. While initial feedback from patients and caretakers has been favorable, the age restriction on KHINDIVI’s label has limited its adoption. The product’s current approved indication is for patients age five and above, but the largest unmet need for precision dosing and liquid dosage is in the infant and toddler population under five years old. In September, Eton met with the FDA to discuss a pathway for the company’s revised KHINDIVI formulation with lower levels of inactive ingredients. The agency indicated an openness to expanding the indicated age and requested the Company conduct a bioequivalence study comparing the two formulations. The bioequivalence study is scheduled to initiate by January 2026 , which would allow for the revised formulation to be submitted as a supplement to the existing NDA in the second quarter of 2026. The FDA indicated a 10-month review for the submission, leading to a potential early 2027 approval. The Company expects product adoption to significantly increase if KHINDIVI is approved for younger patients. NDA for ET-600 accepted for review by the FDA. In July, ET-600’s NDA was accepted for review by the FDA and assigned a PDUFA target action date of February 25, 2026. The Company has scheduled the production of launch inventory for the fourth quarter in preparation for a commercial launch shortly after the anticipated approval. Pre-launch marketing activities are underway. The Company recently held an ET-600 advisory meeting with leading healthcare practitioners within the pediatric endocrinology community, and patients and healthcare providers continue to express excitement for the product. Third Quarter Financial Results Net Revenue: Net revenues for the third quarter of 2025 were $22.5 million compared with $10.3 million in the prior year period, an increase of 118%. Net product revenues for the third quarter of 2025 were $22.5 million compared with $9.8 million in the prior year period, an increase of 129%. The growth was driven primarily by increased sales of ALKINDI SPRINKLE and Carglumic Acid and the addition of sales from INCRELEX and GALZIN. $0.9 million of product revenue was from the sale of finished product inventory to Ipsen S.A. (“Ipsen”) and Esteve Pharmaceuticals, S.A. (“Esteve”) and transition services to facilitate the ownership transition of INCRELEX in certain European countries and is expected to be non-recurring. $2.4 million of revenue was derived from an initial loading order of semi-finished INCRELEX inventory to Esteve. The Company previously entered into a long-term supply agreement with Esteve and expects such semi-finished good purchases to continue on an annual or semi-annual basis. Gross Profit: Gross profit for the third quarter of 2025 was $7.9 million compared with $6.3 million in the prior year period, an increase of 25%. Gross profit was negatively impacted by costs related to the transition of the ex- U.S. distribution of INCRELEX. Of total cost of goods sold (COGS) expense in the quarter, $1.4 million was due to sales commissions to Ipsen related to transition services. $1.9 million of COGS expense was finished product inventory sold to Ipsen and Esteve to facilitate the ownership transition. Marketing authorizations have now been transferred in key European countries and the company expects transition related costs to decline significantly in the fourth quarter and cease completely by mid-2026. An additional $4.1 million of COGS expense was from the initial loading order of semi-finished goods to Esteve to support future in-market sales. Adjusted gross profit, which adjusts for the impact of acquired inventory step-up adjustments and intangible amortization, was $10.2 million in the third quarter of 2025, representing an adjusted gross margin of 45% compared to adjusted gross profit of $6.6 million and adjusted gross margin of 64% in the prior year period. Adjusted gross margin in the quarter was negatively impacted by INCRELEX ex-US related costs. The Company expects to report fourth quarter adjusted gross margin of approximately 70%. Research and Development (R&D) Expenses: R&D expenses for the third quarter of 2025 were $1.1 million compared to $0.5 million in the prior year period, due primarily to increased expenses associated with the Company’s ET-700 and ET-800 development activities. General and Administrative (G&A) Expenses: G&A expenses for the third quarter of 2025 were $8.1 million compared to $5.3 million in the prior year period, due primarily to an increase in product advertising and launch-year promotional expenses, higher stock-based compensation expense, and an increase in compensation and benefit expenses due to an increase in general and administrative headcount. Adjusted G&A expense, which removes share-based compensation, transaction-related costs, and other one-time expenses, was $6.9 million in the quarter, compared with $4.3 million the prior year period. The Company expects Adjusted G&A expenses to remain flat or decline in the fourth quarter of 2025. Adjusted Earnings Before Interest, Taxes, Depreciation and Amortization (Adjusted EBITDA): Adjusted EBITDA for the third quarter of 2025 was $2.9 million and was negatively impacted by the INCRELEX ex-US related costs. Adjusted EBITDA was $2.0 million in the prior year period. Net Income/Loss: Net loss for the third quarter of 2025 was $1.9 million or $0.07 per diluted share compared to net income of $0.6 million or $0.02 per diluted share in the prior year period. On a non-GAAP basis, the Company reported net income of $1.5 million or $0.04 per diluted share, for the third quarter of 2025 compared to net income of $1.9 million , or $0.07 per diluted share in the prior year period. For a reconciliation of GAAP net loss to Earnings Before Interest, Taxes, Depreciation and Amortization EBITDA (“EBITDA”), Adjusted EBITDA and adjusted Non-GAAP basic and fully diluted earnings per share to the most directly comparable GAAP financial measure, please see the tables below. Cash Position: As of September 30, 2025 , the Company had cash and cash equivalents of $37.1 million and generated $12.0 million of operating cash flow during the quarter. During the quarter, Eton received a $4.3 million cash payment for the INCRELEX ex- U.S. licensing agreement executed in the first quarter. Conference Call and Webcast Information As previously announced, Eton Pharmaceuticals will host its third quarter 2025 conference call as follows: In addition to taking live questions from participants on the conference call, management will be answering emailed questions from investors. Investors can email questions to: investorrelations@etonpharma.com. The live webcast can be accessed on the Investors section of Eton’s website at https://ir.etonpharma.com/. An archived webcast will be available on Eton’s website approximately two hours after the completion of the event and for 30 days thereafter. * Conference call participants should register to obtain their dial-in and passcode details. Please be sure to register using a valid email address. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has eight commercial rare disease products: KHINDIVI™, INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has five additional product candidates in late-stage development: ET-600, Amglidia®, ET- 700, ET -800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Non-GAAP Financial Measures In addition to the Company’s results of operations determined in accordance with U.S. generally accepted accounting principles (GAAP), which are presented and discussed above, management also utilizes Adjusted EBITDA, an unaudited financial measure that is not calculated in accordance with GAAP, to evaluate the Company’s financial results and performance and to plan and forecast future periods. Adjusted EBITDA is considered a “non-GAAP” financial measure within the meaning of Regulation G promulgated by the SEC . Management believes that this non-GAAP financial measure reflects an additional way of viewing aspects of the Company’s operations that, when viewed with GAAP results, provides a more complete understanding of the Company’s results of operations and the factors and trends affecting its business. Management believes Adjusted EBITDA provides meaningful supplemental information regarding the Company’s performance because (i) it allows for greater transparency with respect to key metrics used by management in its financial and operational decision-making; (ii) it excludes the impact of non-cash or, when specified, non-recurring items that are not directly attributable to the Company’s core operating performance and that may obscure trends in the Company’s core operating performance; and (iii) it is used by institutional investors and the analyst community to help analyze the Company’s results. However, Adjusted EBITDA and any other non-GAAP financial measures should be considered as a supplement to, and not as a substitute for, or superior to, the corresponding measures calculated in accordance with GAAP. Further, non-GAAP financial measures used by the Company and the way they are calculated may differ from the non-GAAP financial measures or the calculations of the same non-GAAP financial measures used by other companies, including the Company’s competitors. Adjusted EBITDA The Company defines Adjusted EBITDA as net loss, excluding the effects of stock-based compensation and expenses, interest, taxes, depreciation, amortization, investment loss, net, and, if any and when specified, other non-recurring income or expense items. Management believes that the most directly comparable GAAP financial measure to Adjusted EBITDA is net loss. Adjusted EBITDA has limitations and should not be considered as an alternative to gross profit or net loss as a measure of operating performance or to net cash provided by (used in) operating, investing, or financing activities as a measure of ability to meet cash needs. Investor Relations: Lisa M. Wilson , In-Site Communications, Inc. T: 212-452-2793E: lwilson@insitecony.com (1) Represents depreciation expense related to our property and equipment. (2) Intangible amortization expenses are associated with our intellectual property rights related to INCRELEX®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous and Nitisinone. (3) During the three and nine months ended September 30, 2025 , we recognized in cost of sales $1,324 and $3,673 , respectively, for inventory step-up expense primarily attributable to INCRELEX® inventory revalued in connection with this product acquisition. (4) Represents share-based compensation expense associated with our stock option and restricted stock unit stock unit grants to our employees and non-employee directors and our employee share purchase plan. (5) Represents severance and benefit expenses associated with role redundancy within commercial operations during the first quarter of 2025. (6) Represents legal expense and other divestiture-related costs associated with the out-licensing of the INCRELEX® commercial rights in territories outside of the U.S. (7) Income tax adjustments on pre-tax non-GAAP adjustments represent the estimated income tax impact of each pre-tax non-GAAP adjustment based on the effective income tax rate for the period as the Company is in a full income tax valuation allowance position and the income tax adjustments on a pre-tax non-GAAP adjustment is commensurate with the performance measure. Source: Eton Pharmaceuticals

Financial StatementAcquisitionDrug ApprovalNDA

16 Oct 2025

·www.businesswire.com

Cycle Pharmaceuticals Launches Its First Oncology Product, PHYRAGO™ (dasatinib) Tablets, in the US

DETROIT--(BUSINESS WIRE)--Cycle Pharmaceuticals announces the launch of PHYRAGO, its first oncology product in the US. PHYRAGO is launched in partnership with Handa Therapeutics, LLC, and will be exclusively available through specialty pharmacy Onco360® with a specialty distribution network from McKesson, Cencora, and Cardinal Health. Cycle Pharmaceuticals launches its first oncology product, PHYRAGO™ (dasatinib) tablets, in the US PHYRAGO is a tyrosine kinase inhibitor indicated for the treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML), adult patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL), and pediatric patients 1 year of age and older with Ph+ CML in chronic phase, and newly diagnosed Ph+ ALL.1 PHYRAGO is bioequivalent to Sprycel® (dasatinib) tablets,2 and has the unique clinical benefit of allowing patients to take proton pump inhibitors (PPIs) and H2 receptor antagonists (H2RAs) concomitantly to help manage gastric acid disorders.1 To help patients with medication access, financial savings, and clinical support, Cycle Vita™ will be available to eligible* patients taking PHYRAGO.† “We are honored to introduce PHYRAGO as a new treatment option for people living with Ph+ leukemia,” said Steve Fuller, Chief Strategy Officer at Cycle Pharmaceuticals. “We understand the daily challenges that come with managing Ph+ CML and Ph+ ALL, and our goal is to ease that burden, not just with an improved formulation of dasatinib, but through the individualized care and hands on support offered by our Cycle Vita program. By enabling more flexibility in co-medication and providing a dedicated support team, we’re committed to helping patients and their families navigate treatment with greater confidence and peace of mind.” Cycle Pharmaceuticals has been empowering patients with rare diseases through its products and support hub since 2017. NITYR ® (nitisinone) Tablets in 2017 SAJAZIR™ (icatibant) Injection in 2021 JAVYGTOR™ (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution in 2022 TASCENSO ODT ® (fingolimod) in 2023 ORMALVI™ (dichlorphenamide) tablets in 2024 VENXXIVA™ (tiopronin) Delayed-Release Tablets in 2025 BAFIERTAM ® (monomethyl fumarate) Delayed-Release Capsules in 2025 HARLIKU™ (nitisinone) Tablets in 2025 Indications PHYRAGO™ is a kinase inhibitor indicated for the treatment of: Newly diagnosed adults with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase. Adults with chronic, accelerated, or myeloid or lymphoid blast phase Ph+ CML with resistance / intolerance to prior therapy including imatinib. Adults with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL) with resistance / intolerance to prior therapy. Pediatric patients 1 year of age and older with Ph+ CML in chronic phase. Pediatric patients 1 year of age and older with newly diagnosed Ph+ ALL in combination with chemotherapy. Important Safety Information Warnings and Precautions: Myelosuppression and Bleeding Events: Severe thrombocytopenia, neutropenia and anemia may occur. Use caution if used concomitantly with medication that inhibits platelet function or anticoagulants. Monitor blood counts. Transfuse and interrupt PHYRAGO when indicated. Fluid Retention: Fluid retention, including pleural effusions. Manage with supportive care and/or dose modification. Cardiovascular Toxicity: Monitor for signs or symptoms and treat appropriately. Pulmonary Arterial Hypertension (PAH): PHYRAGO may increase the risk of developing PAH which may be reversible on discontinuation. Consider baseline risk and evaluate patients for signs and symptoms of PAH during treatment. Stop PHYRAGO if PAH is confirmed. QT Prolongation: Use PHYRAGO with caution in patients who have / may develop prolongation of the QT interval. Severe Dermatologic Reactions: Severe mucocutaneous dermatologic reactions have been reported. Tumor Lysis Syndrome: Tumor lysis syndrome has been reported. Maintain hydration and correct uric acid levels prior to initiating treatment. Embryo-Fetal Toxicity: Can cause fetal harm. Advise patients of reproductive potential, of potential risk to fetus and to use effective contraception. Effects on Growth and Development in Pediatric Patients: Epiphyses delayed fusion, osteopenia, growth retardation, and gynecomastia have been reported. Monitor bone growth and development. Hepatotoxicity: Assess liver function before treatment, monthly thereafter or as clinically indicated. Monitor liver function when combined with chemotherapy. Adverse Reactions: Common adverse reactions in adults include myelosuppression, fluid retention events, diarrhea, headache, skin rash, hemorrhage, dyspnea, fatigue, nausea, and musculoskeletal pain. Common adverse reactions in pediatric patients include mucositis, febrile neutropenia, pyrexia, diarrhea, nausea, vomiting, musculoskeletal pain, abdominal pain, cough, headache, rash, fatigue, constipation, arrhythmia, hypertension, edema, infections (bacterial, viral and fungal), hypotension, decreased appetite, hypersensitivity, dyspnea, epistaxis, peripheral neuropathy, and altered state of consciousness. Postmarketing Experience: Post approval adverse reactions: Hepatitis B virus reactivation, atrial fibrillation/atrial flutter, interstitial lung disease, chylothorax, Stevens-Johnson syndrome, nephrotic syndrome, thrombotic microangiopathy, hepatotoxicity. Drug Interactions: Strong CYP3A4 Inhibitors and Strong CYP3A4 Inducers: Dose reduction may be necessary. Antacids: Avoid concomitant use. Use in Specific Populations: Pregnancy: PHYRAGO can cause fetal harm. Advise a pregnant woman of the potential risk to a fetus. Contraception: Should be used during treatment and for 30 days after the last dose. Lactation: Breastfeeding is not recommended during treatment and for 2 weeks after the last dose. Pediatric Use: There is no data in children under 1 year of age. Adverse reactions associated with bone growth and development were reported and should be monitored closely. Refer to the full USPI for pediatric patients with difficulty swallowing tablets. Geriatric Use: Patients over 65 and older may experience: fatigue, pleural effusion, diarrhea, dyspnea, cough, lower gastrointestinal hemorrhage, appetite disturbance, abdominal distention, dizziness, pericardial effusion, congestive heart failure, hypertension, pulmonary Cinzia, and weight decrease. Monitor closely. For more detailed information, please refer to the full Prescribing Information at phyrago.com/PI/. To report SUSPECTED ADVERSE REACTIONS, contact Cycle Pharmaceuticals at 1-844-784-1807, or the FDA at: 1-800-FDA-1088 or www.fda.gov/medwatch. US-DAS-2500114 (October 2025) References PHYRAGO™ (dasatinib). Prescribing Information. Handa Therapeutics, LLC. Study NC9013-002. Center for Drug Evaluation and Research – Clinical Pharmacology Review(s). 2022. [online]. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/nda/2024/216099Orig1s000ClinPharmR.pdf . [Accessed May 21 2025]. *Some areas of support may not be accessible to all patients. Eligibility criteria may apply to ensure compliance with all applicable federal and state requirements, and benefits may be limited to commercially insured patients only. For more detailed information about eligibility, terms and conditions, please contact the Cycle Vita team at 888-360-8482. †Patients who do not wish to have Cycle Vita hub support may be eligible for Co-Pay. -ENDS- ©2025 Cycle Pharmaceuticals Limited. All rights reserved. PHYRAGO™ is a trademark of Handa Therapeutics, LLC. Sprycel® is a registered trademark of Bristol-Myers Squibb Company. NITYR® and TASCENSO ODT® are registered trademarks of Cycle Pharmaceuticals Ltd. in the United States. Cycle Vita™, SAJAZIR™, JAVYGTOR™, and HARLIKU™ are trademarks of Cycle Pharmaceuticals Ltd. in the United States. ORMALVI™ and VENXXIVA™ are trademarks of Torrent Pharma Inc. in the United States. BAFIERTAM® is a registered trademark of Banner Life Sciences, LLC, a company of Cycle Pharmaceuticals. About Cycle Pharmaceuticals Cycle Pharmaceuticals was founded in 2012 with the sole aim of delivering drug treatments and product support to the underserved rare disease community. Cycle Pharma focuses on rare genetic conditions in metabolic, immunology, urology, and oncology. In neurology, we focus on multiple sclerosis. Cycle Pharma is headquartered in Cambridge, UK and has offices in Detroit, Michigan and Boston, Massachusetts. For more information, please visit www.cyclepharma.com and follow us on X, LinkedIn and Facebook. About Handa Pharmaceuticals, Inc. Handa is a specialty pharmaceutical company that focuses on developing high-quality medications that address significant unmet medical needs in oncology and neurology. Handa’s leadership has a proven track record of building effective teams, high quality product portfolios and successful pharmaceutical businesses. For more information, please visit www.handapharma.com. About Onco360 Oncology Pharmacy Onco360 is the nation’s largest independent Oncology Pharmacy and clinical support services company. Onco360 was founded in 2003 to bring together the stakeholders involved in the cancer treatment process and serve the specialized needs of oncologists, patients, hospitals, cancer centers of excellence, manufacturers, health plans, and payers. It dispenses nationally through its network of URAC-, and ACHC-accredited Oncology Pharmacies. Onco360 is headquartered in Louisville, Kentucky, and is a flagship specialty pharmacy brand of PharMerica Corporation, a leading institutional pharmacy, specialty infusion, and hospital services company servicing healthcare facilities in the United States. For more information about Onco360, please visit Onco360.com.

Drug Approval

14 Oct 2025

·www.einpresswire.com

NHS to screen all newborn babies for life-threatening metabolic disorder

Newborn babies will now be routinely screened and treated earlier on the NHS for a rare, life-threatening metabolic disorder, which can result in the need for a liver transplant. Hereditary Tyrosinaemia Type 1 (HT1) is a rare, genetically inherited disorder that affects around 7 babies per year in the UK. Left untreated, it can lead to severe complications such as organ damage and liver failure. This condition will now be screened for, in the blood test that babies get on the fifth day after they are born, taken from the heel. The test will help to identify and treat babies before symptoms appear, meaning they can get life-saving daily drug treatment sooner and be placed on a restricted diet to make them less likely to face long-term health problems in later life. HT1 affects the way in which the body breaks down protein in food. Protein is usually broken down into amino acids by enzymes. In people affected by HT1, there is a mutation to the gene that controls the enzyme which breaks down an amino acid called tyrosine. When the body is unable to break down tyrosine, a harmful chemical builds up in the blood; and left untreated, the disorder can cause liver failure, kidney disease and can lead to liver cancer. The NHS has added HT1 to the NHS Newborn Blood Spot Screening Programme in England, following a recommendation by the UK National Screening Committee. Once identified by screening, babies can be placed on a medication called Nitisinone and a special diet of regulated amounts of normal milk feed (breast or formula milk) with a special milk that is low in tyrosine. Older children with HT1 have a special low-tyrosine diet that will be managed by specialist dietitians. The combination of Nitisinone and tyrosine-restricted diet can prevent the long-term complications of HT1 from developing. Dulcie-May and her partner Ryan live in Bridgewater with their two-year-old-old daughter, Darla-May, and 15-month-old son, Hugo. Soon after Darla-May was born, her mum noticed she had an enlarged stomach. They went to the doctor and to A&E, but her condition wasn’t diagnosed straight away. When Darla-May was 10 months old, doctors told the family her liver, spleen and kidneys felt enlarged. She was sent for an emergency cancer referral and a stomach scan and blood tests showed that her liver was failing and her blood wasn’t clotting. She was transferred to Birmingham Children’s Hospital where a liver specialist and metabolic team diagnosed Hereditary Tyrosinemia type 1 (HT1). She was placed on medication and a low protein diet and is now thriving back at home. Dulcie-May said : “Seeing our daughter in pain was horrible. She was in such a bad way, and to not know when we were going home or if she would react well to the medication was just heartbreaking. “Now Darla-May is thriving. Not long after coming home she started to crawl, and now she’s a very happy toddler, and to look at her you wouldn’t think she has a rare metabolic genetic disorder “Ultimately, screening for tyrosinaemia will save lives. I think it’s so important that the NHS offers it. I also think it will help families feel less alone. They’ll be reassured to know that if they’re planning on having more children in future the condition will be caught straight away.” Dr Harrison Carter, NHS Director of Vaccination and Screening, said: “Being able to screen for tyrosinaemia will help give hundreds of thousands of families extra reassurance and peace of mind – and while rare genetic conditions will be ruled out in most cases, for those families affected it means treatment and care can begin straight away, to improve their baby’s chances of leading a healthy life. “This is a really vital step forward in care for newborn babies and will be important news for families who might be at risk of potentially life-threatening hereditary genetic conditions.” NHS Chief Midwife Kate Brintworth said: “The arrival of a newborn baby can be a very exciting and busy time for families, but the blood spot test is a key part of early NHS care and support because it can tell families so much about their baby’s future health. “Women and families can find out more about the test from their midwife during their pregnancy, including the different conditions the test is screening for, so we’d encourage them to talk to their maternity team about it.” Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

100 Deals associated with Nitisinone

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KEGG	Wiki	ATC	Drug Bank
D05177	Nitisinone	A16AX04	DB00348

R&D Status

10 top approved records.

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Indication	Country/Location	Organization	Date
Alkaptonuria	European Union	Swedish Orphan Biovitrum International AB	21 Feb 2005
Alkaptonuria	Iceland	Swedish Orphan Biovitrum International AB	21 Feb 2005
Alkaptonuria	Liechtenstein	Swedish Orphan Biovitrum International AB	21 Feb 2005
Alkaptonuria	Norway	Swedish Orphan Biovitrum International AB	21 Feb 2005
Tyrosinemias	European Union	Swedish Orphan Biovitrum International AB	21 Feb 2005
Tyrosinemias	Iceland	Swedish Orphan Biovitrum International AB	21 Feb 2005
Tyrosinemias	Liechtenstein	Swedish Orphan Biovitrum International AB	21 Feb 2005
Tyrosinemias	Norway	Swedish Orphan Biovitrum International AB	21 Feb 2005
Tyrosinemia, Type I	United States	Swedish Orphan Biovitrum AB	18 Jan 2002

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT00107783 (FDA_CDER) Manual	Phase 2	Alkaptonuria	40	HARLIKUHARLIKU 2 mg	iubmcxjqnd(wcmowifikq) = smclfwrxmk hyvmawmnib (knvslxigty, -79 to -97) View more	Positive	10 Jun 2025
				No Treatment	iubmcxjqnd(wcmowifikq) = qquulhuuqh hyvmawmnib (knvslxigty, 0 - +216) View more
NCT01916382 (SONIA 2, ESC2024)	Phase 4	Aortic Valve Stenosis \| Alkaptonuria homogentisic acid levels	138	Nitisinone	zvqhehffsw(vdjapwdisj) = zxfokxmlwk nkdclzlunw (vtxluuqfni, -0.054 to 0.18)	Positive	30 Aug 2024
NCT01390077 (CTgov)	Phase 2/3	Alkaptonuria	8	Nitisinone	yujksgougt(ehrwwrnxie) = smkxpgprkh vtzxlukgaz (tqzhwgncqg, owffykxhbg - zvfdnotuwi) View more	-	22 Apr 2021
NCT01916382 (SONIA 2, Pubmed \| Lancet Diabetes Endocrinol) Manual	Phase 3	Alkaptonuria	138	nitisinone	jttksjsnca(tgeacfolhu) = xbbmaixkws budpzaykcr (jucgmordub ) View more	Positive	01 Sep 2020
				no treatment	shpaqlbbmt(jfmvkgqsay) = qwmslqfqjc saictdvhfu (culhovptuu )
NCT01838655 (Pubmed \| JCI Insight)	Phase 1/2	Oculocutaneous Albinism Type 1B	5	Nitisinone 2 mg/d	aknuqhfbtr(ucmscowgqk) = ugrdwrgatd rsojolndkx (fgmygazniq, ± 1.54) View more	-	24 Jan 2019
NCT01838655 (CTgov)	Phase 1/2	Blindness \| Oculocutaneous Albinism Type 1B	5	Nitisinone	arolqixytr(brvdomujxh) = kpyunrtsrj ffvbezrvhh (wgteimxpoq, 1.68) View more	-	17 Nov 2017
NCT02750709 (CTgov)	Phase 1	Tyrosinemia, Type I	24	Nitisinone (Treatment Period 1)	sdfhfyqkzs(uufrldwlmu) = trwgvrlldw feioabijsn (fziddlagdx, 17.9) View more	-	13 Dec 2016
				Nitisinone (Treatment Period 2)	sdfhfyqkzs(uufrldwlmu) = rkufqgktct feioabijsn (fziddlagdx, 24.2) View more
NCT02750332 (CTgov)	Phase 1	Tyrosinemia, Type I	20	Nitisinone (Test Product (Fasted))	neflvjtuxl(zkrqwptvif) = iwahrqzkzn uaxxvfuqyh (nsozsgvosb, 22.2) View more	-	13 Dec 2016
				Nitisinone (Test Product (Fed))	neflvjtuxl(zkrqwptvif) = cjkgfopkwp uaxxvfuqyh (nsozsgvosb, 15.6) View more
NCT02750345 (CTgov)	Phase 1	Tyrosinemia, Type I	24	Nitisinone (Test Product 1)	dvedttugee(tyudelagol) = nwabodwdnv xirdwpaegk (xdlpjuzige, 17.8) View more	-	17 Oct 2016
				Nitisinone (Test Product 2)	dvedttugee(tyudelagol) = bbmcfijufe xirdwpaegk (xdlpjuzige, 16.6) View more
NCT01857362 (CTgov)	Phase 1	-	12	Nitisinone (Nitisinone 2 x 10 mg Capsules)	rkmghkfuvr(zzyoqcasgd) = wisadjzrtm wxxhspwnmm (csivijnvyx, muoeyjyiwv - sbnisxebzy) View more	-	27 Oct 2014
				Nitisinone (Nitisinone 1 x 20 mg Capsules)	rkmghkfuvr(zzyoqcasgd) = dnhmecarrp wxxhspwnmm (csivijnvyx, cvbsdgkayh - trlhxoaaze) View more

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