Phase III, Single Arm, Open Label, International, Multi Centre Study to Evaluate the Efficacy and Safety of Lomitapide in Paediatric Patients With Homozygous Familial Hypercholesterolaemia (HoFH) on Stable Lipid Lowering Therapy

This is a single arm, open label, multi centre phase III study to evaluate the efficacy and long term safety of lomitapide in paediatric patients with HoFH receiving stable LLT (including LA, when applicable) comprising of the following phases:
Screening Period (starting at Week 12, i.e. ≤12 weeks prior to Baseline for up to 6 weeks)
Stratified Enrolment and Start of Run in Period (starting at minimum at Week 6, i.e., 6 weeks prior to Baseline for a minimum of 6 weeks):
Efficacy Phase (starting at Baseline, i.e. Day [D] 0 for 24 weeks±3 days
Safety Phase (starting at Week 24±3 days for 80±1 weeks)

Start Date14 Dec 2020

Sponsor / Collaborator

Amryt Pharma Holdings Ltd.

EUCTR2019-002278-30-IT / Unknown statusPhase 3

Phase III, single-arm, open-label, international, multi-centre study to evaluate the efficacy and safety of lomitapide in paediatric patients with Homozygous Familial Hypercholesterolaemia (HoFH) on stable lipid-lowering therapy - APH-19

Start Date11 Nov 2020

Sponsor / Collaborator

Amryt Pharmaceuticals DAC

EUCTR2018-002911-80-IT / Unknown statusPhase 3

Open-label study to evaluate the safety, tolerability, and efficacy of LOmitapide for the treatment of patients with Familial CHylomicroNEmia Syndrome - LOCHNES

Start Date18 Dec 2018

Sponsor / Collaborator-

100 Clinical Results associated with Lomitapide Mesylate

100 Translational Medicine associated with Lomitapide Mesylate

100 Patents (Medical) associated with Lomitapide Mesylate

225

Literatures (Medical) associated with Lomitapide Mesylate

01 Feb 2024·European journal of pharmacology

Drug-repurposing by virtual and experimental screening of PFKFB3 inhibitors for pancreatic cancer therapy

Article

Author: Duan, Hong-Quan ; Yu, Yang ; Zhong, Zhi-Xin ; Qin, Nan ; Jiang, Xiao ; Li, Xiao-Lei ; Cao, Xin ; Liu, Lu ; Chen, Ying ; Duan, Xiao-Chuan ; Ni, Tian-Wen ; Li, Xu-Zhao

Pancreatic cancer (PC) is the most frequently occurring cancer, with few effective treatments and a 5-year survival rate of only about 11%. It is characterized by stiff interstitium and pressure on blood vessels, leading to an increased glycolytic metabolism. PFKFB3 plays an important role in glycolysis, and its products (fructose-2,6-bisphosphate), which are allosteric PFK1 activators, limit the glycolytic rate. In this study, 14 PFKFB3 inhibitors were obtained by virtually screening the FDA-approved compound library. Subsequently, the in-vitro investigations confirmed that Lomitapide and Cabozantinib S-malate exhibit the excellent potential to inhibit PFKFB3. The combined administration of Lomitapide and Gemcitabine at a certain molar ratio indicated an enhanced anti-tumor effect in Orthotopic Pancreatic Cancer (OPC) models. This investigation provides a new treatment strategy for PC therapy.

30 Jan 2024·Circulation

Evinacumab for Pediatric Patients With Homozygous Familial Hypercholesterolemia

Article

Author: Mendell, Jeanne ; Reijman, M Doortje ; Wiegman, Albert ; George, Richard T ; Hartz, Jacob ; Baker-Smith, Carissa ; Charng, Min-Ji ; Brinton, Eliot A ; Srinivasan, Shubha ; Bihorel, Sébastien ; Banerjee, Poulabi ; Ali, Shazia ; Greber-Platzer, Susanne ; Baum, Seth ; Pordy, Robert ; Hirshberg, Boaz ; Moriarty, Patrick M ; Brothers, Julie A

BACKGROUND::

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder characterized by severely elevated low-density lipoprotein cholesterol (LDL-C) levels due to profoundly defective LDL receptor (LDLR) function. Given that severely elevated LDL-C starts in utero, atherosclerosis often presents during childhood or adolescence, creating a largely unmet need for aggressive LDLR-independent lipid-lowering therapies in young patients with HoFH. Here we present the first evaluation of the efficacy and safety of evinacumab, a novel LDLR-independent lipid-lowering therapy, in pediatric patients with HoFH from parts A and B of a 3-part study.

METHODS::

The phase 3, part B, open-label study treated 14 patients 5 to 11 years of age with genetically proven HoFH (true homozygotes and compound heterozygotes) with LDL-C >130 mg/dL, despite optimized lipid-lowering therapy (including LDLR-independent apheresis and lomitapide), with intravenous evinacumab 15 mg/kg every 4 weeks.

RESULTS::

Evinacumab treatment rapidly and durably (through week 24) decreased LDL-C with profound reduction in the first week, with a mean (SE) LDL-C reduction of −48.3% (10.4%) from baseline to week 24. ApoB (mean [SE], –41.3% [9.0%]), non–high-density lipoprotein cholesterol (–48.9% [9.8%]), and total cholesterol (–49.1% [8.1%]) were similarly decreased. Treatment-emergent adverse events were reported in 10 (71.4%) patients; however, only 2 (14.3%) reported events that were considered to be treatment-related (nausea and abdominal pain). One serious treatment-emergent adverse event of tonsillitis occurred (n=1), but this was not considered treatment-related.

CONCLUSIONS::

Evinacumab constitutes a new treatment for pediatric patients with HoFH and inadequately controlled LDL-C despite optimized lipid-lowering therapy, lowering LDL-C levels by nearly half in these extremely high-risk and difficult-to-treat individuals.

REGISTRATION::

URL: https://www.clinicaltrials.gov ; Unique identifier: NCT04233918.

01 Dec 2023·The Canadian journal of cardiology

New Biological Therapies for Low-Density Lipoprotein Cholesterol

Review

Author: Gill, Praneet K ; Hegele, Robert A

Depressed low-density lipoprotein cholesterol concentration protects against atherosclerotic cardiovascular disease. Natural hypocholesterolemia states can have a monogenic etiology, caused by pathogenic loss of function variants in the PCSK9, ANGPTL3, MTTP, or APOB genes. In this focused review, we discuss development and clinical use of several new therapeutics that inhibit these gene products to target elevated levels of low-density lipoprotein cholesterol. In particular, inhibitors of proprotein convertase subtilisin kexin type 9 (PCSK9) have notably affected clinical practice, followed recently by inhibition of angiopoietin-like 3 (ANGPTL3). Currently used in the clinic are alirocumab and evolocumab, two anti-PCSK9 monoclonal antibodies, inclisiran, a small interfering RNA that prevents PCSK9 translation, evinacumab, an anti-ANGPTL3 monoclonal antibody, and lomitapide, a small-molecule inhibitor of microsomal triglyceride transfer protein. Additional therapies are in preclinical or clinical trial stages of development. These consist of other monoclonal antibodies, antisense oligonucleotides, small-molecule inhibitors, mimetic peptides, adnectins, vaccines, and gene-editing therapies. Vaccines and gene-editing therapies in particular hold great potential to confer active long-term attenuation or provide single-treatment life-long knock-down of PCSK9 or ANGPTL3 activity. Biologic therapies inspired by monogenic hypocholesterolemia states are becoming valuable tools to help protect against atherosclerotic cardiovascular disease.

News (Medical) associated with Lomitapide Mesylate

04 Jan 2024

·www.globenewswire.com

Ultragenyx Receives Positive Recommendation from NICE in the U.K. for Evkeeza® ▼ (evinacumab) for Adolescents and Adults Aged 12 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

NOVATO, Calif., Jan. 04, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the National Institute for Health and Care Excellence (NICE) has issued a final draft guidance recommending Evkeeza® (evinacumab) to NHS England. Evkeeza is recommended within its marketing authorization as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adults and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for this rare and debilitating condition. "We are very pleased that NICE has approved the use of evinacumab within the NHS. Our patients with HoFH often have early onset heart disease, in many cases in their teens, and many of the usual medicines we use to treat cholesterol have limited effectiveness in this condition,” stated Dr. Jaimini Cegla, clinical lead of the Lipid and Cardiovascular Risk Service, Hammersmith Hospital. “Evinacumab, which is effective at lowering LDL-C in HoFH when combined with other lipid-lowering therapies, is a much needed and very welcome addition to help us treat our patients as best we can.” NICE deemed that, despite uncertainties in the cost-effectiveness evidence comparing evinacumab with lomitapide in adults with HoFH, overall there are cost savings with evinacumab compared with lomitapide. The NICE committee acknowledged that clinical trial evidence shows that Evkeeza, combined with other lipid-lowering therapies (LLTs), can lower LDL-C levels when statins and other LLTs have not reduced them enough. NICE also acknowledged that adolescent patients with HoFH on LLTs with or without lipoprotein apheresis have a high unmet need because LLTs have limited effectiveness in this patient population, lipoprotein apheresis can be traumatic and time consuming, and there is potential for an inequality of access if made available to adults and not adolescents. NICE considered that Evkeeza addresses an unmet need for new treatments to lower LDL-C, and that Evkeeza may improve adherence to treatment because it is administered once monthly (every 4 weeks) as an infusion. “We are delighted that patients with HoFH will have another tool to help manage their condition, especially as it is approved from age 12,” stated Jules Payne, CEO of Heart UK. "This recognition from NICE on the clinical and economic benefits of Evkeeza is a meaningful milestone for people living with HoFH in England and Wales,” said Erik Harris, chief commercial officer at Ultragenyx. “This highlights our commitment to working expeditiously on innovative ways to make Evkeeza broadly available to the HoFH community.” Evkeeza is now reimbursed and commercially available to prescribe for appropriate patients with HoFH in the U.S., England and Wales, Canada, Italy and Germany. About Homozygous Familial Hypercholesterolemia (HoFH) HoFH is a devastating form of inherited hypercholesterolemia, affecting 1 in 300,000 people globally and approximately 1,600 people in the European Union. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels (>400 mg/dL) of LDL-C, or bad cholesterol. Patients with HoFH are at risk of premature atherosclerotic disease and cardiac events at an early age. About Evkeeza® (evinacumab) Evinacumab, the active substance in Evkeeza, binds to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood. Evkeeza is delivered via an infusion every 4 weeks. Evkeeza is approved by the Medicines and Healthcare products Regulatory Agency (MHRA) as an adjunct to diet and other low-density lipoprotein-cholesterol (LDL-C) lowering therapies for the treatment of adult and adolescent patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. Regeneron Pharmaceuticals, Inc. discovered and developed Evkeeza and commercializes the product in HoFH in the U.S. under the generic name evinacumab-dgnb, with dgnb as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA. Ultragenyx is responsible for commercialization efforts for Evkeeza in HoFH in countries outside of the U.S. About Ultragenyx Pharmaceutical Inc. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com. Ultragenyx Forward-Looking Statements and Use of Digital Media Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx's expectations and projections regarding its future operating results and financial performance, anticipated cost or expense reductions, the timing, progress and plans for its clinical programs and clinical studies, future regulatory interactions, and the components and timing of regulatory submissions, are forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause the Company’s clinical development programs, commercial success of its products and product candidates, continued collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, risks related to reliance on third-party partners to conduct certain activities on the Company’s behalf; the potential for any license or collaboration agreement, including Ultragenyx’s collaboration agreement with Regeneron to commercialize Evkeeza outside of the United States, to be terminated; uncertainty and potential delays related to clinical drug development; uncertainties and unpredictability of obtaining regulatory approval for the Company’s product candidates and the scope of such potential regulatory approval; smaller than anticipated market opportunities for the Company’s products and product candidates; fluctuations in buying or distribution patterns by distributors and specialty pharmacies; competition to the Company’s products and product candidates; potential undesirable or serious side effects from the Company’s products or product candidates; the Company’s ability to effectively manage the expansion of its commercial organization; market acceptance of the Company’s current or future products; uncertainties related to insurance coverage and reimbursement status of newly approved products; manufacturing risks and supply chain disruptions; competition from other therapies or products; and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the Company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and product candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 3, 2023, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/). ContactsUltragenyx Pharmaceutical Inc.InvestorsJoshua Higa+1-415-475-6370ir@ultragenyx.com MediaCarolyn Wang+1-415-225-5050media@ultragenyx.com

Drug ApprovalLicense out/in

20 Dec 2023

·www.fiercepharma.com

After Amryt buyout, Chiesi scores with FDA nod for rare skin disease treatment Filsuvez

The FDA's approval of rare skin disease drug Filsuvez gives Chiesi Global Rare Diseases its third nod for a new drug from the U.S. regulator this year. Seven months after Krystal Biotech became the first company to gain FDA approval to treat the rare, devastating skin disease epidermolysis bullosa (EB), Chiesi Farmaceutici has followed suit. On Tuesday, the U.S. regulator gave a thumbs up to Chiesi’s Filsuvez (birch triterpenes) to treat both junctional epidermolysis bullosa (JEB) and dystrophic epidermolysis bullosa (DEB) in patients 6 months and older. While Krystal’s topical gene therapy Vyjuvek was endorsed by the FDA in May for DEB, Filsuvez becomes the first FDA-sanctioned treatment for both JEB and DEB. The topical gel treats the wounds associated with EB, a genetic, connective tissue disease that causes the skin to become fragile. Even the slightest friction can tear the skin and cause blisters. EB can be life-threatening as patients are at risk of developing aggressive squamous cell carcinomas and infections. The active ingredient in Filsuvez, which is found in birch trees, accelerates healing and reduces the size of the wound. “The FDA’s decision to approve Filsuvez provides those living with EB a safe and effective treatment option for the most prominent and difficult symptom of EB, open wounds that may not heal,” said Brett Kopelan, the executive director of debra of America, a non-profit that raises money and supports those with EB. This is the third FDA approval this year for Chiesi Global Rare Diseases, which was established in 2020 as one three business units of the family-owned Chiesi Group. The other FDA nods came for enzyme replacement therapies Elfabrio, which treats the neurological disorder Fabry disease, and Lamzede for the genetic condition alpha-mannosidosis, which causes a buildup of sugars in the organs. The FDA green light for Filsuvez was based on the phase 3 EASE trial, which showed significant increase in healing and wound closure compared to patients who received an active control gel, measured at 45 days. Filsuvez was rejected by the FDA in February of 2022, with the regulator asking for more efficacy data. Four months later, Europe endorsed the treatment for DEB and JEB. The drug was owned then by Amryt Pharma of Ireland. In January of this year, Chiesi acquired Amryt Pharma in a $1.25 billion buyout. Along with the purchase came three approved rare disease treatments—Juxtapid, for hypercholerterolemia, Myalept, for lipodystrophy, and Mycapssa, for acromegaly. With its launch of Filsuvez, Chiesi will face formidable competition. Krystal Biotech's launch of Vyvuvek has been so promising that ISI Evercore bumped up its projected 2024 sales of the gene therapy to $273 million.

AcquisitionPhase 3Drug ApprovalClinical ResultGene Therapy

19 Dec 2023

·www.globenewswire.com

Ultragenyx Receives European Commission Decision for Evkeeza® (evinacumab) Expanded Indication in Children Aged 5 Years and Older with Homozygous Familial Hypercholesterolemia (HoFH)

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that the European Commission (EC) has extended the approval of Evkeeza® (evinacumab) as an adjunct to diet and other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH. Evkeeza initially received EC decision as an adjunct to diet and other lipid-lowering therapies in adolescents and adults aged 12 years and older with HoFH in June 2021. “This approval heralds a new era for treatment of children 5 to 11 years of age with HoFH,” stated Albert Wiegman, M.D., Ph.D. and Professor, Department of Paediatrics at Amsterdam University Medical Center. “The addition of Evkeeza to aggressive baseline lipid-lowering therapy nearly halves LDL-C levels in a disorder with an extremely high risk of premature cardiovascular disease in young patients, where achieving LDL-C goals otherwise isn’t possible. This EC decision follows the positive recommendation from the Committee for Medicinal Products for Human Use received in November 2023 and is based on the results of a Phase 3 open-label study in patients ages 5-11 years with HoFH.1 Despite treatment with other lipid-lowering therapies, children entered the Phase 3 trial with an average LDL-C level of 264 mg/dL, more than twice the target (<110 mg/dL) for paediatric patients with HoFH. With the addition of Evkeeza, children (n=14) were able to reduce their LDL-C by 48% at week 24 on average. Significant reductions were also observed in other key secondary endpoints including levels of apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol (non-HDL-C) and total cholesterol. The safety profile of Evkeeza was consistent with the safety profile observed in adults and paediatric patients aged 12 years and older, with the additional adverse reaction of fatigue (reported in 3 patients). “It is with immense joy that the international community of parents and caregivers of children with HoFH welcomes this approval, because Evkeeza is a potentially life-changing therapy for the children and their parents affected by this rare and severe disorder,” stated Magdalena Daccord, chief executive officer of FH Europe Foundation. “As we advocate for childhood screening and detection to help improve early HoFH diagnosis, it is key to be able to offer to young patients appropriate and innovative treatment solutions along with lifestyle management. That said, it will be a true success once this therapy option is available to all those who need it as soon as they need it to support their treatment goals.” Evkeeza is delivered via 60-minute intravenous infusion once monthly (every 4 weeks). The treatment is now reimbursed and commercially available to prescribe for appropriate patients with HoFH in the U.S., Canada, Italy and Germany. It is also available via early access schemes in Austria and France. "It is critical to reduce LDL levels as early as possible for all people living with HoFH especially given that children as young as 5 years of age are at risk for severe cardiac events that can be life threatening,”2 said Eric Crombez, M.D., chief medical officer at Ultragenyx. “With its novel mechanism, Evkeeza combined with other lipid-lowering therapies has demonstrated the ability to significantly reduce LDL cholesterol levels beyond historical standard of care, which could have a transformational impact for these younger patients.” The three-part, single-arm, open-label trial evaluated Evkeeza added to other lipid-lowering therapies in paediatric patients with HoFH aged 5 to 11 years. Part A (n=6) was a Phase 1b trial designed to assess the pharmacokinetics (PK), safety and tolerability of Evkeeza. Part B (n=14) evaluated the efficacy of Evkeeza during a 24-week treatment period and enrolled patients with an average age of 9 years. Among them, 86% were on statins, 93% were on ezetimibe, 50% were on LDL apheresis and 14% were on lomitapide. Patients received Evkeeza 15 mg/kg every four weeks delivered intravenously in addition to their baseline lipid-lowering treatment regimen. The primary endpoint was change in LDL-C at week 24. Secondary endpoints included the effect of Evkeeza on other lipid parameters (i.e., apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein[a] and total cholesterol), efficacy by mutation status, safety and tolerability, immunogenicity and PK. Patients who completed Part A or B were allowed to, and all did, continue treatment in Part C (n=20), a completed Phase 3 extension trial. This trial was not designed to evaluate the effect of Evkeeza on cardiovascular events. Evkeeza was well tolerated in this patient population. The most common adverse events (AEs) occurring in >15% of patients were COVID-19 (n=15), pyrexia (n=5), headache (n=4), throat pain (oropharyngeal pain, n=4) as well as upper abdominal pain, diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis and cough (all n=3). Most reported AEs were mild or moderate, and none led to study discontinuation. HoFH is a devastating form of inherited hypercholesterolemia, affecting 1 in 300,000 people globally and approximately 1,600 people in the European Union. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels (>400 mg/dL) of LDL-C, or bad cholesterol. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events at an early age. Evinacumab, the active substance in Evkeeza, attaches to a protein in the body called ANGPTL3 and blocks its effects. ANGPTL3 is involved in controlling cholesterol levels and blocking its effect reduces the level of cholesterol in the blood. Evkeeza is delivered via an infusion every month (4 weeks). Evkeeza is approved by the European Commission (EC) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of paediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH). The effects of Evkeeza on cardiovascular morbidity and mortality have not been determined. Regeneron Pharmaceuticals, Inc. discovered and developed Evkeeza, and commercializes the product in HoFH in the U.S. under the generic name evinacumab-dgnb, with dgnb as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA. Ultragenyx is responsible for commercialization efforts for Evkeeza in countries outside of the U.S. The content above comes from the network. if any infringement, please contact us to modify.

Clinical ResultPhase 3Drug Approval

100 Deals associated with Lomitapide Mesylate

External Link

KEGG	Wiki	ATC	Drug Bank
D09638	Lomitapide Mesylate	C10AX12	DB08827

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Hyperlipidemia Type IIa	MX	University of Pennsylvania	09 Feb 2014
Homozygous familial hypercholesterolemia	US	CHIESI Farmaceutici SpA	21 Dec 2012

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hyperlipoproteinemia Type I	Phase 3	IT	Amryt Pharma Holdings Ltd.	12 Dec 2018
Hyperlipoproteinemia Type II	Phase 3	US	Aegerion Pharmaceuticals, Inc.	29 Oct 2009
Hyperlipoproteinemia Type II	Phase 3	CA	Aegerion Pharmaceuticals, Inc.	29 Oct 2009
Hyperlipoproteinemia Type II	Phase 3	IT	Aegerion Pharmaceuticals, Inc.	29 Oct 2009
Hyperlipoproteinemia Type II	Phase 3	ZA	Aegerion Pharmaceuticals, Inc.	29 Oct 2009
Hyperlipidemias	Phase 2	US	Aegerion Pharmaceuticals, Inc.	01 Oct 2007
Hypercholesterolemia	Phase 2	US	Aegerion Pharmaceuticals, Inc.	01 Apr 2007

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


EUCTR2018-002911-80-IT (LOCHNES, Pubmed) Manual	Not Applicable	Familial chylomicronaemia syndrome	18	lomitapide	ujkhrvoepn(snpsdkjwuk) = zkmtgrrsie zjyogybxpw (pkyakemrai )	Positive	10 Sep 2022
NCT01915771 (CTgov)	Phase 1	-	15	Lomitapide (PK of Lomitapide)	ejkeinbksu(sadrarstgx) = tfeiwtledw gzamxavzrh (mpdxbhykil, ekuewsanlt - fftvwinynl) View more	-	11 Mar 2020
				Lomitapide (PK of M1)	ejkeinbksu(sadrarstgx) = gmbqqprpau gzamxavzrh (mpdxbhykil, fymxealzqm - fzyrbpodcu) View more
ESC2019	Not Applicable	Homozygous familial hypercholesterolemia	12	Lomitapide	tzrjqfxfik(ifsniebyfn) = Two patients stopped lomitapide due to diet and alcohol restrictions ejvekhcfvd (hqiwsajqar ) View more	-	03 Sep 2019
				LL drugs + lipoprotein apheresis
NCT00943306 (CTgov)	Phase 3	Homozygous familial hypercholesterolemia \| Hyperlipoproteinemia Type II	19	lomitapide	pyktudlolh(ctyzsjsyio) = xfuqoptnar pbexqbwkcc (iqhcgjnkul, wwsddttrwy - jjzkiyzurb) View more	-	11 May 2018
NCT02044419 (CTgov)	Phase 1	-	32	Lomitapide (Lomitapide Sprinkled in Applesauce (Treatment A))	ysavxmvofn(irilfqqiss) = cpsaetbxqm xzlhnokkph (sagmrnrpxs, naztoukedl - yfydtaavrk) View more	-	13 Feb 2018
				Lomitapide (Lomitapide Sprinkled in Mashed Banana (Treatment B))	ysavxmvofn(irilfqqiss) = hgzruvwfqt xzlhnokkph (sagmrnrpxs, dipatsbhio - rdcepsyclj) View more
NCT00730236 (Phase 3, Pubmed \| Atherosclerosis)	Phase 3	Homozygous familial hypercholesterolemia	29	Lomitapide	wwpdfftljd(ytqezyfqow) = vktoaduywc dmdicsinte (pswrynlelg )	Positive	01 Jun 2015
				lomitapide (Lipoprotein apheresis)	wwpdfftljd(ytqezyfqow) = tsjvadeoli dmdicsinte (pswrynlelg )
NCT00690443 (CTgov)	Phase 2	Hypercholesterolemia	44	Atorvastatin (Atorvastatin 20 mg)	kezcnylbka(bbhyjsnckj) = fkiznhfenj qhiuoxnydp (tkcpxnbmjm, mqnyflfbwr - dxxqvdmlvn) View more	-	25 Feb 2013
				Lomitapide+Atorvastatin (Atorvastatin 20 mg + Lomitapide)	kezcnylbka(bbhyjsnckj) = ylrgftjxpx qhiuoxnydp (tkcpxnbmjm, cmzzgkleyd - zgrgozinyt) View more
NCT01556906 (CTgov)	Phase 2	Homozygous familial hypercholesterolemia	6	Lomitapide	xlvtmznlew(etavddcstg) = bsjmdoxsvh ptjynzhndg (mnurokndxc, qzsvwrvqoq - dmxlzojykt) View more	-	22 Feb 2013
NCT00730236 (CTgov)	Phase 3	Homozygous familial hypercholesterolemia	29	Lomitapide	rsxroryakw(yphgnagisq) = rwbjfwcilz norjtxxizd (avckistlhh, bozagyckrg - sixjtslcet) View more	-	22 Feb 2013
NCT00559962 (CTgov)	Phase 2	Hyperlipidemias	260	placebo (Placebo)	evuqseojms(levsiqxjup) = zfbstlbwmr xwiidhjpjx (yveqqbhqri, veepacgenp - jgifrhuaaq) View more	-	22 Feb 2013
				AEGR-733 (AEGR-733 5 mg)	evuqseojms(levsiqxjup) = rnclnsgtkn xwiidhjpjx (yveqqbhqri, sxmuqekppm - mhmngysdpv) View more

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