A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients From Birth to 11Years of Age With Primary Hyperoxaluria and Relatively Intact Renal Function

The aim of this study is to evaluate nedosiran in participants 11 years of age and younger who have Primary Hyperoxaluria with relatively intact renal function.

Start Date22 Feb 2022

Sponsor / Collaborator

Dicerna Pharmaceuticals, Inc.

LBCTR2021104866 / SuspendedPhase 2

A Phase 2 Open-Label Multicenter Study to Evaluate the Safety, Pharmacokinetics, and Efficacy of Nedosiran in Pediatric Patients from Birth to 5 Years of Age with Primary Hyperoxaluria and Relatively Intact Renal Function

Start Date06 Dec 2021

Sponsor / Collaborator

Dicerna Pharmaceuticals, Inc.

EUCTR2021-001083-16-IT / Unknown statusPhase 2

Start Date27 Oct 2021

Sponsor / Collaborator

Dicerna Pharmaceuticals, Inc.

100 Clinical Results associated with Nedosiran

100 Translational Medicine associated with Nedosiran

100 Patents (Medical) associated with Nedosiran

Literatures (Medical) associated with Nedosiran

01 Dec 2023·Clinical pharmacology in drug development

Safety, Pharmacokinetics, and Exposure–Response Modeling of Nedosiran in Participants With Severe Chronic Kidney Disease

Article

Author: Zhang, Steven ; Amrite, Aniruddha ; Marbury, Thomas C ; Fuentes, Ernesto

Abstract:

Nedosiran is an investigational RNA‐interference therapeutic in development for primary hyperoxaluria (PH). Because nedosiran undergoes renal clearance, we assessed its pharmacokinetic profile in non‐PH participants with normal kidney function and Stages 4/5 chronic kidney disease (CKD), the latter with/without dialysis. Nedosiran exposure–response modeling in patients with PH Subtype 1 (PH1) with different renal function level was performed to recommend a nedosiran dose for this subpatient population. In this open‐label, single‐dose, Phase 1 study, 24 participants with estimated glomerular filtration rate <30 mL/min/1.73 m2 (CKD Stages 4/5; on hemodialysis [Groups 1a, 1b] and not on hemodialysis [Group 2]) and 10 participants with normal kidney function (estimated glomerular filtration rate ≥90 mL/min/1.73 m2; Group 3) received a single dose of subcutaneous nedosiran sodium 170 mg. Group 1a received nedosiran 8 hours before beginning hemodialysis, Group 1b received nedosiran 2 hours after completing hemodialysis; Group 2 was not on hemodialysis. Nedosiran population pharmacokinetic–pharmacodynamic analyses were conducted using pooled data from this study and 4 others. Nedosiran pharmacokinetic exposure in non‐PH participants with CKD Stages 4/5 was approximately 2‐fold higher versus participants with normal kidney function. Hemodialysis timing relative to nedosiran administration had no clinically significant impact on pharmacokinetics (Group 1a vs 1b). Nedosiran was well tolerated. Modeling indicated that in patients with PH1 with CKD Stages 4/5, lower nedosiran doses provide similar exposure and potential reduction in 24‐hour urinary oxalate to standard nedosiran doses in patients with PH1 with normal kidney function or CKD Stages 2/3. Nedosiran dosage reductions are recommended in patients with PH1 with CKD Stages 4/5; further adjustments are unnecessary if dialysis is started.

01 Dec 2023·Drugs

Nedosiran: First Approval

Review

Author: Syed, Yahiya Y

Nedosiran (RIVFLOZA™), a once-monthly subcutaneous small interfering RNA (siRNA) therapy, is being developed by Dicerna Pharmaceuticals, a Novo Nordisk company, for the treatment of primary hyperoxaluria (PH). It reduces oxalate overproduction by inhibiting the expression of the hepatic lactate dehydrogenase (LDH) enzyme. Nedosiran received its first approval on 29 September 2023 in the USA to lower urinary oxalate levels in children aged ≥ 9 years and adults with PH type 1 (PH1) and relatively preserved kidney function [e.g. estimated glomerular filtration rate (eGFR) ≥ 30 mL/min/1.73 m²]. This article summarizes the milestones in the development of nedosiran leading to this first approval for PH1.

01 Jan 2023·Kidney international

PHYOX2: a pivotal randomized study of nedosiran in primary hyperoxaluria type 1 or 2

Article

Author: Zhou, Jing ; Ariceta, Gema ; Hayes, Wesley ; Wolley, Martin ; Amrite, Aniruddha ; Cochat, Pierre ; Takayama, Tatsuya ; Russell, Kerry ; Groothoff, Jaap ; Belostotsky, Vladimir ; Langman, Craig ; Forbes, Thomas A ; Moochhala, Shabbir H ; Rosskamp, Ralf ; Hamamoto, Shuzo ; Torres, Armando ; Mourani, Chebl ; Lieske, John C ; Hoppe, Bernd ; Satoh, Hiroyuki ; Tönshoff, Burkhard ; Baum, Michelle A

Nedosiran is an investigational RNA interference agent designed to inhibit expression of hepatic lactate dehydrogenase, the enzyme thought responsible for the terminal step of oxalate synthesis. Oxalate overproduction is the hallmark of all genetic subtypes of primary hyperoxaluria (PH). In this double-blind, placebo-controlled study, we randomly assigned (2:1) 35 participants with PH1 (n = 29) or PH2 (n = 6) with eGFR ≥30 mL/min/1.73 m² to subcutaneous nedosiran or placebo once monthly for 6 months. The area under the curve (AUC) of percent reduction from baseline in 24-hour urinary oxalate (Uox) excretion (primary endpoint), between day 90-180, was significantly greater with nedosiran vs placebo (least squares mean [SE], +3507 [788] vs -1664 [1190], respectively; difference, 5172; 95% CI 2929-7414; P < 0.001). A greater proportion of participants receiving nedosiran vs placebo achieved normal or near-normal (<0.60 mmol/24 hours; <1.3 × ULN) Uox excretion on ≥2 consecutive visits starting at day 90 (50% vs 0; P = 0.002); this effect was mirrored in the nedosiran-treated PH1 subgroup (64.7% vs 0; P < 0.001). The PH1 subgroup maintained a sustained Uox reduction while on nedosiran, whereas no consistent effect was seen in the PH2 subgroup. Nedosiran-treated participants with PH1 also showed a significant reduction in plasma oxalate versus placebo (P = 0.017). Nedosiran was generally safe and well tolerated. In the nedosiran arm, the incidence of injection-site reactions was 9% (all mild and self-limiting). In conclusion, participants with PH1 receiving nedosiran had clinically meaningful reductions in Uox, the mediator of kidney damage in PH.

News (Medical) associated with Nedosiran

20 Mar 2024

·www.fiercepharma.com

#FierceMadness is back: Get voting in the 2024 drug name tournament

This year we’re taking the branded drug names that were officially approved by the FDA in 2022 and 2023 - now it's your turn to vote for your winner. LAUNCHED: Wednesday, March 20, 7:00 a.m. ET It’s March, so it’s time for some #FierceMadness. Last year, we channeled the NCAA tournament for the best pharma ad campaigns, but, this year, we’re looking for the best of the best in drug names. Here’s how it works. We’ve chosen 64 of the most distinctive drug names to come out of the FDA’s 2022 and 2023 crop of new approvals. Whittling down to 64 from the 92 drug names available was our staff challenge, but now it’s up to you, readers, to deliberate and vote down to the final winner. Remember, this isn’t about the drugs themselves—neither how well they may work (or not) nor any controversies in how they may have been approved. Our goal is to assess their marketing suitability, considering how well they perform in their respective field and against competitors. Ready to play? Check out the bracket here (PDF) and see the full Madness 64 list below. Then vote is via our poll here. You can vote from now until Friday, March 22, at 6 p.m. ET. Check back here Monday, March 25, for your results, and the chance to vote in the next round. After that, we’ll be back with a new round each Wednesday and Monday through Monday, April 8, when you can vote for your championship winner. Any remote robo-voting will be noted, and those votes will be discarded. Please play fair. Don’t forget to tell us why you picked what you picked in the voting poll comments section—and what you think each drug name really sounds like. We’ll include the funniest and most out-of-the-box comments in our recaps every round, so make 'em good! New this year: Our sister publication Fierce Biotech is also running its own Madness tournament for the best of the best in biotech company names. Check out and vote in its championship here. Good luck, and happy Madness! #FierceMadness 2024: The full bracket of 64 Jesduvroq vs. Lamzede Jesduvroq Maker: GSK Indication: Anemia due to chronic kidney disease Sounds like: When you hit your finger with a hammer, but it’s in front of your children so you’re trying not to swear Lamzede Maker: Chiesi Indication: Non-central nervous system manifestations of alpha-mannosidosis Sounds like: A vegan meal Pluvicto vs. Lunsumio Pluvicto Maker: Novartis Indication: Prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer following other therapies Sounds like: A new planet Lunsumio Maker: Roche Indication: Relapsed or refractory follicular lymphoma, a type of non-Hodgkin lymphoma Sounds like: A Japanese wrestler Briumvi vs. Camzyos Briumvi Maker: TG Therapeutics Indication: Relapsing forms of multiple sclerosis Sounds like: A sixth-generation cheese Camzyos Maker: Bristol Myers Squibb Indication: Obstructive hypertrophic cardiomyopathy Sounds like: A Spanish holiday resort Voquezna vs. Mounjaro Voquezna Maker: Phathom Pharmaceuticals Indication: Helicobacter pylori infection Sounds like: A sports drink with a kick Mounjaro Maker: Eli Lilly Indication: Lowering blood sugar levels in Type 2 diabetes Sounds like: A legendary medieval sword fighter Vtama vs. Amvuttra Vtama Maker: Dermavant Indication: Plaque psoriasis Sounds like: A mouthful Amvuttra Maker: Alnylam Indication: Polyneuropathy of hereditary transthyretin-mediated amyloidosis Sounds like: A pope’s gift Xenpozyme vs. Daxxify Xenpozyme Maker: Sanofi Indication: Acid sphingomyelinase deficiency Sounds like: An alien protein Daxxify Maker: Revance Indication: The treatment of cervical dystonia and for the temporary improvement of frown lines Sounds like: A ray gun to make you sassy Fruzaqla vs. Sotyktu Fruzaqla Maker: Takeda Pharmaceuticals Indication: Refractory, metastatic colorectal cancer Sounds like: A European swear word Sotyktu Maker: Bristol Myers Squibb Indication: Moderate to severe plaque psoriasis Sounds like: Gesundheit! Terlivaz vs. Imjudo Terlivaz Maker: Mallinckrodt Pharmaceuticals Indication: Improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function Sounds like: A Hungarian dance Imjudo Maker: AstraZeneca Indication: Unresectable hepatocellular carcinoma Sounds like: A self-confident martial artist Elahere vs. Vabysmo Elahere Maker: ImmunoGen Indication: Recurrent ovarian cancer that is resistant to platinum therapy Sounds like: An inebriated attempt at Hawaiian Vabysmo Maker: Roche Indication: Neovascular (wet) aged-related macular degeneration and diabetic macular edema Sounds like: A new way of saying fantastic Pyrukynd vs. Rystiggo Pyrukynd Maker: Agios Indication: Hemolytic anemia in pyruvate kinase deficiency Sounds like: A drunk Scrabble game Rystiggo Maker: UCB Indication: Generalized myasthenia gravis in adults who are anti-acetylcholine receptor- or anti-muscle-specific tyrosine kinase antibody-positive Sounds like: An Italian village Beyfortus vs. Xdemvy Beyfortus Maker: AstraZeneca Indication: To prevent respiratory syncytial virus lower respiratory tract disease Sounds like: A medieval stronghold Xdemvy Maker: Tarsus Pharmaceuticals Indication: Demodex blepharitis Sounds like: Elon Musk’s drunk attempt at renaming his business Tzield vs. Rezlidhia Tzield Maker: Provention Bio Indication: Delay the onset of stage 3 Type 1 diabetes Sounds like: An electronic barrier Rezlidhia Maker: Rigel Pharmaceuticals Indication: Relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation Sounds like: A new Disney princess Krazati vs. Leqembi Krazati Maker: Mirati Therapeutics Indication: KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer in adults who have received at least one prior systemic therapy Sounds like: A crazy party Leqembi Maker: Eisai Indication: Alzheimer’s disease Sounds like: An inspirational French aphorism Cibinqo vs. Brenzavvy Cibinqo Maker: Pfizer Indication: Refractory, moderate to severe atopic dermatitis Sounds like: A game of cheeky bingo Brenzavvy Maker: TheracosBio Indication: Glycemic control in adults with Type 2 diabetes mellitus as an adjunct to diet and exercise Sounds like: Heard in a copy of "Pirates of the Caribbean" Jaypirca vs. Skyclarys Jaypirca Maker: Eli Lilly Indication: Relapsed or refractory mantle cell lymphoma in adults who have had at least two lines of systemic therapy, including a BTK inhibitor Sounds like: A strong coffee Skyclarys Maker: Biogen Indication: Friedreich’s ataxia Sounds like: A high-rise building Zavzpret vs. Paxlovid Zavzpret Maker: Pfizer Indication: Migraine treatment Sounds like: A Turkish fast-food restaurant Paxlovid Maker: Pfizer Indication: Mild to moderate COVID-19 in adults at high risk for progression to severe COVID-19 Sounds like: A bizarre Pac-Man sequel Inpefa vs. Columvi Inpefa Maker: Lexicon Pharmaceuticals Indication: Heart failure Sounds like: An anti-fascist offshoot group Columvi Maker: Roche Indication: Diffuse large B-cell lymphoma, not otherwise specified, or large B-cell lymphoma arising from follicular lymphoma after two or more lines of systemic therapy Sounds like: An ancient Roman detective Daybue vs. Zynyz Daybue Maker: Acadia Indication: Rett syndrome Sounds like: How my five-year-old insists debut is spelled Zynyz Maker: Incyte Indication: Metastatic or recurrent locally advanced Merkel cell carcinoma Sounds like: Drug naming companies may be running out of ideas Rezzayo vs. Qalsody Rezzayo Maker: Melinta Therapeutics Indication: Candidemia and invasive candidiasis Sounds like: A phrase used when you’ve had a great nap in the day Qalsody Maker: Biogen Indication: Amyotrophic lateral sclerosis in adults who have a SOD1 gene mutation Sounds like: From the makers of “Better Call Saul” comes “better Qalsody” Elfabrio vs. Veozah Elfabrio Maker: Chiesi Indication: Fabry disease Sounds like: The lost Spanish Mario brother Veozah Maker: Astellas Indication: Moderate to severe hot flashes caused by menopause Sounds like: A Broadway play Epkinly vs. Zurzuvae Epkinly Makers: AbbVie/Genmab Indication: Relapsed or refractory diffuse large B-cell lymphoma and high-grade B-cell lymphoma after two or more lines of systemic therapy Sounds like: Someone who watches too many streaming episodes Zurzuvae Makers: Sage/Biogen Indication: Postpartum depression Sounds like: A mouthful Izervay vs. Talvey Izervay Maker: Iveric Bio Indication: Geographic atrophy secondary to age-related macular degeneration Sounds like: A new app for scanning the land Talvey Maker: Johnson & Johnson Indication: Relapsed or refractory multiple myeloma who have received at least four prior therapies Sounds like: A little too close to talc… Elrexfio vs. Veopoz Elrexfio Maker: Pfizer Indication: Relapsed or refractory multiple myeloma who have received at least four prior lines of therapy Sounds like: A new dinosaur found in Mexico Veopoz Maker: Regeneron Pharmaceuticals Indication: Patients 1 year old and older with CD55-deficient protein-losing enteropathy Sounds like: Double-plus good! Zilbrysq vs. Ojjaara Zilbrysq Maker: UCB Indication: Generalized myasthenia gravis in adults who are anti-acetylcholine receptor antibody positive Sounds like: Absolute zero Ojjaara Maker: GSK Indication: Intermediate or high-risk myelofibrosis in adults with anemia Sounds like: “What’s my name?” Exxua vs. Pombiliti Exxua Maker: Fabre-Kramer Pharmaceuticals Indication: Major depressive disorder Sounds like: Someone who’s always leaving Pombiliti Maker: Amicus Indication: Late-onset Pompe disease Sounds like: The ability to treat Pompe disease (this feels a little on the nose) Rivfloza vs. Velsipity Rivfloza Maker: Novo Nordisk Indication: Urinary oxalate levels in patients 9 years and older with primary hyperoxaluria type 1 and relatively preserved kidney function Sounds like: An exuberant DJ Velsipity Maker: Pfizer Indication: Moderately to severely active ulcerative colitis in adults Sounds like: A deodorant brand you wouldn’t want to buy Bimzelx vs. Omvoh Bimzelx Maker: UCB Indication: Moderate to severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy Sounds like: Throwing away unwanted antacids Omvoh Maker: Eli Lilly Indication: Ulcerative colitis Sounds like: A new age prayer Loqtorzi vs. Defencath Loqtorzi Maker: Coherus Biosciences Indication: Recurrent or metastatic nasopharyngeal carcinoma when used together with or following other therapies Sounds like: How you close the door in a magical kingdom Defencath Maker: CorMedix Indication: Reduce the incidence of catheter-related bloodstream infections in adults with kidney failure receiving chronic hemodialysis Sounds like: A Dungeons and Dragons character Augtyro vs. Ryzneuta Augtyro Maker: Bristol Myers Squibb Indication: ROS1-positive non-small cell lung cancer Sounds like: A virtual reality game where you’re the bad guy Ryzneuta Maker: Acrotech Biopharma Indication: Neutropenia Sounds like: Getting rid of someone’s charisma Truqap vs. Ogsiveo Truqap Maker: AstraZeneca Indication: Breast cancer that meets certain disease criteria Sounds like: A limit on truth Ogsiveo Maker: SpringWorks Indication: Progressing desmoid tumors who require systemic treatment Sounds like: Oh, give us a go! Quviviq vs. Ztalmy Quviviq Maker: Idorsia Indication: Insomnia Sounds like: A producer asking a biotech founder turned politician to get ready for his TV appearance Ztalmy Maker: Marinus Indication: Seizures in cyclin-dependent kinase-like 5 deficiency disorder Sounds like: A nefarious Yevgeny Zamyatin character Opdualag vs. Wainua Opdualag Maker: Bristol Myers Squibb Indication: Unresectable or metastatic melanoma Sounds like: A Russian prison Wainua Maker: AstraZeneca Indication: Polyneuropathy of hereditary transthyretin-mediated amyloidosis Sounds like: A Premier League footballer

08 Oct 2023

·medcitynews.com

Takeda to Pull Lung Cancer Drug from Market After Failed Confirmatory Study

Accelerated approval offers a way to more quickly bring patients drugs for diseases that have few treatment options. But that speedy path to the market comes with the understanding that the FDA can take its regulatory blessing away—unless the company takes its drug away first. That is what Takeda Pharmaceutical has decided to do with its cancer drug, Exkivity. Following discussions with the FDA, the Japanese pharmaceutical giant is voluntarily withdrawing Exkivity from the market. Exkivity treats non-small cell lung cancer by targeting epidermal growth factor (EGFR). That cancer protein must have exon 20 mutations—the same genetic signature addressed by Johnson & Johnson’s Rybrevant. As a small molecule in a capsule formulation, Takeda’s drug offered the advantage of oral dosing compared to infused Rybrevant. Both received accelerated approvals in 2021 based on results from open label studies. A drug awarded accelerated approval must confirm its safety and efficacy in a post-marketing study. In July, when Takeda reported financial results for its fiscal 2023 first quarter, the company disclosed that the Exkivity Phase 3 confirmatory study was stopped for futility. Last week’s announcement about the voluntary withdrawal of the drug did not include any details about the study results, but Takeda said full data will be presented at an upcoming medical meeting or published in a peer-reviewed journal. Meanwhile, J&J has filed for full FDA approval of Rybrevant. In July, the company reported that the drug, in combination with chemotherapy, posted Phase 3 results showing statistically significant and clinically meaningful improvement measured against the main goal of progression-free survival. J&J is seeking approval of this drug combination as a first-line treatment for locally advanced or metastatic non-small cell lung cancer carrying EGFR exon 20 insertion mutations. In addition to withdrawing Exkivity in the U.S., Takeda is also working to voluntarily pull the drug from other markets where it is approved. Not all withdrawals happen in such an orderly and timely fashion. The FDA pulled Covis Pharma’s pre-term birth drug Makena from the market in April, four years after that drug failed its confirmatory study. There were plenty of other pharmaceutical regulatory developments in the past week. Here’s a recap of recent regulatory news: —Novo Nordisk’s nedosiran, brand name Rivfloza, received FDA approval for treating primary hyperoxaluria type 1. The rare metabolic disorder is an inherited deficiency of an enzyme needed to break down oxalate, a compound produced by the liver and also found in some foods. The resulting oxalate buildup in the kidneys leads to chronic kidney disease. Rivfloza works by a mechanism called RNA interference, in which small interfering RNA stop the production of a disease-causing protein. The Novo Nordisk drug is intended to reduce oxalate production in the liver. The FDA decision for Rivfloza covers the use of the once-monthly subcutaneous injection in adults as well as children age 9 and older. Novo Nordisk added Rivfloza to its pipeline via the $3.3 billion acquisition of Dicerna Pharma in 2021. The drug will compete against Alnylam Pharmaceuticals’ Oxlumo, which in 2020 became the first FDA-approved therapy for primary hyperoxaluria. The Alnylam drug is a subcutaneous injection administered every three months. —The FDA placed a partial hold on clinical testing of Innate Pharma’s lacutamab following a patient death. A Phase 2 study in cutaneous T cell lymphoma completed enrollment earlier this year. A Phase 1b test in peripheral T cell lymphoma is awaiting an interim analysis. Marseille, France-based Innate said the deceased patient developed hemophagocytic lympohistiocytosis, a rare and severe adverse reaction. No other details were disclosed. The partial hold means patients who have received the study drug and are benefitting from it may continue to receive the treatment after renewing their consent. However, no new patients may be enrolled in the clinical trials until the FDA lifts the partial hold. Lacutamab is an antibody that blocks KIR3DL2, a receptor expressed in about 65% of cutaneous T cell lymphoma subtypes and about 90% of patients with aggressive forms of this cancer. —Novavax’s updated Covid-19 vaccine was granted emergency use authorization for those age 12 and older. The decision amends last year’s FDA authorization and makes the vaccine the only protein-based Covid-19 vaccine available in the U.S. Authorization of the updated Novavax Covid-19 vaccine comes a month after the FDA approved updated mRNA Covid-19 vaccines from Moderna and partners Pfizer and BioNTech. —The FDA declined to approve Eli Lilly’s lebrikizumab as a treatment for moderate-to-severe atopic dermatitis. According to the pharma giant, the agency cited findings from an inspection of the site of a third-party manufacturer. No issues were raised about the safety or efficacy of the antibody drug. Lilly said it will work with the manufacturer and the FDA to address the issues raised by the regulator. The lebrikizumab rejection follows the April negative regulatory decision for ulcerative colitis drug mirikizumab. In that decision, the agency also cited manufacturing issues. —Manufacturing issues were also cited in the FDA rejection of relaotulinumtoxinA, a product that Swiss company Galderma developed for treating moderate-to-severe glabellar lines (frown lines) associated with a muscle that contracts the skin into wrinkles. The product is also a potential treatment for canthal lines (crow’s feet). Galderma said it has identified changes the manufacturing process to address the FDA’s feedback. Regulatory filings in other markets are ongoing and remain on track. —FDA approval of Biogen’s Tofidence made that drug the first biosimilar to Roche inflammatory disease drug Actemra. Tofidence may now be used for treating rheumatoid arthritis in adults; polyarticular juvenile idiopathic arthritis in those age 2 and older; and systemic juvenile idiopathic arthritis in those age 2 and older. —The FDA approved Ocuphire Pharma’s Ryzumvi, a drug that reverses the effects of eye-dilating drops. Pharmacologically induced eye dilation leads to light sensitivity and difficulty reading and driving—effects that can last up to 24 hours. Ryzumvi is administered as one to two drops in each eye following a patient’s eye exam. The approval covers adults and children 12 and older. Ocuphire developed the drug in partnership with Viatris. —Leqembi, an Alzheimer’s therapy developed by Eisai and Biogen, is now approved in Japan, making it the second country to approve the intravenously infused antibody drug. Japan’s regulatory decision comes just shy of three months after the FDA awarded full approval to the product. Photo by FDA

Drug ApprovalVaccinePhase 3Clinical ResultAccelerated Approval

02 Oct 2023

·www.fiercepharma.com

With FDA nod for rare disease drug Rivfloza, Novo Nordisk eyes showdown with Alnylam

Novo Nordisk got its hands on Rivfloza in its 2021 buyout of Dicerna Pharmaceuticals. Novo Nordisk has spent the last few years grappling with the demands stemming from the launch of its popular obesity therapy Wegovy. Now, with a new FDA approval, the drugmaker is making a splash in a much rarer disease. Novo’s Rivfloza, a treatment for the rare disease primary hyperoxaluria type 1 (PH1), has gained approval from the FDA, the company said Monday. A monthly, subcutaneous treatment, Rivfloza belongs to the of ribonucleic acid interference (RNAi) class of drugs. It’s the second FDA-approved option for the disease after Alnylam’s rival RNAi therapy Oxlumo won its FDA approval in November 2020. PH1 is a rare, severe disease characterized by an overproduction of oxalate, which causes the formation of kidney stones and leads to progressive kidney damage. It’s estimated about 2,000 patients live with the disease in the U.S., Novo said. Many are believed to be undiagnosed. The Danish drugmaker got its hands on the therapy with its 2021 purchase of Dicerna Pharmaceuticals for $3.3 billion. At the time, the drug was Dicerna’s lead pipeline candidate. Dicerna previously studied the drug in other forms of primary hyperoxaluria (PH2 and 3), but ultimately narrowed its focus to PH1 after seeing early data. The FDA endorsed Rivfloza to lower urinary oxalate levels in PH1 patients 9 and older who have relatively preserved kidney function. In a phase 2 study, patients on the medicine experienced a reduction in urinary oxalate excretion compared with placebo, according to Novo. As for rival Alnylam, the company has been busy with launch efforts for Oxlumo since its 2020 approval. By the end of June, more than 350 commercial patients were taking the drug worldwide. In the second quarter, the drug generated $24.2 million. Oxlumo is given subcutaneously every three months, while Novo’s Rivfloza is a monthly therapy. Novo plans to launch its drug in early 2024.

Drug ApprovalAcquisitionClinical ResultPhase 2

100 Deals associated with Nedosiran

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Primary Hyperoxaluria Type 1	US	Novo Nordisk, Inc.	29 Sep 2023

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Primary Hyperoxaluria Type 2	Phase 3	US	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	JP	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	AU	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	CA	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	FR	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	DE	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	IT	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	LB	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	NL	Dicerna Pharmaceuticals, Inc.	09 Jul 2019
Primary Hyperoxaluria Type 2	Phase 3	NO	Dicerna Pharmaceuticals, Inc.	09 Jul 2019

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03847909 (PHYOX2, CTgov)	Phase 2	Primary Hyperoxaluria Type 2 \| Primary Hyperoxaluria Type 1	35	DCR-PHXC	uojvdvjoyv(ooynehtnhi) = fxmcfltbdh tcbibkkeqf (lkkukpzhrm, gnuxnriler - soskugrdpc) View more	-	22 May 2024
NCT03847909 (PHYOX2, FDA) Manual	Phase 2	Primary Hyperoxaluria Type 1	35	RIVFLOZA	jyyxfbdxek(wsizfozfto) = oxsvruaihk hzdgwthamu (hbqmhqbnqd, -5025 to -1947) View more	Positive	29 Sep 2023
NCT03847909 (PHYOX2, FDA) Manual	Phase 2	Primary Hyperoxaluria Type 1	35	Placebo	jyyxfbdxek(wsizfozfto) = tmolnrnxgk hzdgwthamu (hbqmhqbnqd, 781 - 3761) View more	Positive	29 Sep 2023
NCT04042402 (PHYOX3, ASN2022)	Phase 3	Primary Hyperoxaluria Type 1 LDHA gene	-	Nedosiran	fvtljnjbng(bmldbcxgzw) = There were no drug-related SAEs or study discontinuations, or deaths fwddbnuvns (xondbgefca ) View more	Positive	03 Nov 2022
NCT04042402 (PHYOX3, ASN2022)	Phase 3	Primary Hyperoxaluria Type 1 LDHA gene	-	Placebo		Positive	03 Nov 2022
compassionate use (ASN2021)	Not Applicable	Primary Hyperoxaluria Type 1 Maintenance	2	Pyridoxine	vodeebjbcr(jnvygmdtzr) = uzywikbjaj bjgqosotql (amweoyzegr )	Positive	27 Oct 2021
NCT04555486 (PHYOX™4 \| PHYOX4, BusinessWire) Manual	Phase 1	Primary Hyperoxaluria Type 2	6	Nedosiran	qnigenikpp(oavdwkcpzt) = all AEs were mild and unrelated，cAE was back pain，no SAE qysmwzfxat (jdjsxwnkex ) View more	Positive	19 Oct 2021
NCT04555486 (PHYOX™4 \| PHYOX4, BusinessWire) Manual	Phase 1	Primary Hyperoxaluria Type 2	6	Placebo		Positive	19 Oct 2021
NCT03392896 (PHYOX1, Pubmed) Manual	Phase 1	Hyperoxaluria, Primary	43	nedosiran (Group A；healthy participants)	eefgeqcqxt(ueutxionar) = rffgvavizs gczqybvgap (haphvkwzzj )	Positive	02 Sep 2021
NCT03392896 (PHYOX1, Pubmed) Manual	Phase 1	Hyperoxaluria, Primary	43	nedosiran (Group B；patients with PH1 or PH2)	eefgeqcqxt(ueutxionar) = gbbmelffys gczqybvgap (haphvkwzzj ) View more	Positive	02 Sep 2021
MP47-17 (AUA2021)	Not Applicable	Primary Hyperoxaluria Type 1	-	Nedosiran	mcrttrxsfb(lwmdyfeioo) = ufqpjxhmwu wpqlvnazns (bgsreiymrn )	-	01 Sep 2021
NCT04042402 (PHYOX™3 \| PHYOX3, BusinessWire) Manual	Phase 3	Hyperoxaluria, Primary	16	Nedosiran	zsdwjqyvvt(cvlfgudhoo) = khhtbsufeu tsvdvjzlsj (vwbeoilowo ) View more	Positive	22 Oct 2020
NCT04042402 (PHYOX3, ASN2020)	Phase 3	Primary Hyperoxaluria Type 2 genetically confirmed	16	Nedosiran	hfpwnraedq(hqsugtcisy) = Treatment-emergent adverse events (AEs) were observed in 11 participants. Seven participants experienced 33 AEs considered related to study drug: administration-site events (18), blood chemistry findings (6), pain (2), dysuria (1), nasal congestion (1), edema (1), and erectile dysfunction (1). Three AEs were uncoded at this time. None of the participants experienced injection-site reactions (defined as occurring 4 hr or more after injection). All drug-related AEs were mild. There were no drug-related serious AEs. dulssbqooo (buzussrhsp ) View more	Positive	19 Oct 2020
NCT03392896 (PHYOX, ASN2019)	Phase 1	Primary Hyperoxaluria Type 2	-	DCR-PHXC 6 mg/kg	twveenrnwy(nvvexvjoru) = ckohssfyny oouqaqtvgo (qcrzaebngl )	Positive	05 Nov 2019

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