Amicus Therapeutics
🇺🇸United States
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2002-01-01
- Employees
- 517
- Market Cap
- $3.3B
- Website
- http://www.amicusrx.com
- Introduction
Amicus Therapeutics, Inc. operates as a biotechnology company. The firm focuses on discovering, developing and delivering medicines for people living with metabolic diseases. Its product portfolio includes first and only approved oral precision medicine for people living with Fabry disease who have amenable genetic variants, a novel, clinical-stage, treatment paradigm for Pompe disease and a rare disease gene therapy portfolio. The company was founded by John Francis Crowley on February 4, 2002 and is headquartered in Princeton, NJ.
Amicus Therapeutics Reports 15% Revenue Growth in Q1 2025, Expands Portfolio with DMX-200 Licensing Deal
• Amicus Therapeutics reported Q1 2025 total revenue of $125.2 million, representing 15% year-over-year growth at constant exchange rates, driven by strong patient demand for both Galafold and Pombiliti + Opfolda.
• The company has expanded its rare disease portfolio by in-licensing DMX-200, a Phase 3 treatment for focal segmental glomerulosclerosis (FSGS), a rare kidney disease with no approved therapies and significant market potential.
• Despite adjusting 2025 revenue growth guidance to 15-22%, Amicus remains on track to achieve GAAP profitability in the second half of 2025 and anticipates surpassing $1 billion in total sales by 2028.
FDA Confirms Proteinuria as Primary Endpoint for Dimerix's DMX-200 Phase 3 FSGS Trial
• The FDA has endorsed proteinuria as an acceptable primary endpoint for full marketing approval of Dimerix's DMX-200 in treating Focal Segmental Glomerular Sclerosis, potentially expediting the path to market.
• Dimerix's DMX-200 has previously demonstrated positive effects on proteinuria in both pre-clinical and clinical studies, strengthening the significance of this regulatory development.
• The FDA remains open to discussing endpoints that could support a potential Accelerated Approval application, offering hope for patients with this progressive kidney disease who have limited treatment options.
Navigating the Orphan Drug Landscape in Europe: HTA Challenges and the Rising Role of Real-World Evidence
• European orphan drug approvals have grown significantly, with 192 approvals through 2022, yet approximately 95% of rare diseases still lack approved treatments, highlighting persistent market access challenges despite regulatory progress.
• HTA outcomes for orphan drugs vary dramatically across European countries, with approval rates ranging from 98% in Germany to 67% in Scotland, creating inconsistent patient access and requiring tailored market entry strategies.
• Real-world evidence (RWE) is emerging as a critical solution to bridge the evidence gap in orphan drug development, with pharmaceutical companies increasingly investing in RWE infrastructure to support regulatory approvals and strengthen value propositions.
Biotech Deal Landscape: February-March 2025 Sees Surge in Partnerships Across Multiple Therapeutic Areas
• The first quarter of 2025 witnessed significant biotech partnership activity, with Eli Lilly, AstraZeneca, and Novo Nordisk emerging as top collaborators in deals worth billions across small molecules, antibodies, and RNA therapeutics.
• February 2025 featured notable acquisitions including Novartis's $2.15 billion buyout of Anthos Therapeutics, while March saw AstraZeneca acquire Belgian biotech EsoBiotec and Bristol Myers Squibb purchase 2seventy bio for $286 million.
• Obesity therapeutics gained significant traction in March 2025, with AbbVie entering the field through a $350 million upfront deal with Gubra for an amylin analog, while Roche partnered with Zealand Pharma on petrelintide in a deal worth up to $5.25 billion.
FDA Approves Vyjuvek: Breakthrough Topical Gene Therapy for Dystrophic Epidermolysis Bullosa
• The FDA has granted approval for Vyjuvek, making it the first-ever topical gene therapy authorized for treating dystrophic epidermolysis bullosa (DEB), a rare genetic condition causing severe skin fragility.
• In pivotal clinical trials, Vyjuvek demonstrated remarkable efficacy with 65% of treated wounds achieving complete healing compared to 26% in the placebo group, offering new hope for DEB patients.
• Krystal Biotech plans to launch Vyjuvek in Q3 at $24,250 per vial, with typical annual treatment costs estimated at $631,500, addressing a condition that currently burdens US families with monthly wound care costs of up to $10,000.
Amicus Therapeutics to Present Extensive Research on Fabry and Pompe Diseases at WORLDSymposium 2025
• Amicus Therapeutics will showcase 16 presentations at WORLDSymposium 2025, featuring comprehensive research on migalastat for Fabry disease and cipaglucosidase alfa plus miglustat for Pompe disease.
• Key studies include long-term patient outcomes from the FollowME Fabry Pathfinders registry and real-world evidence of treatment effectiveness in both rare diseases.
• Notable research highlights treatment satisfaction, quality of life improvements, and clinical outcomes in patients switching from existing therapies to Amicus' novel treatment approaches.
Corcept Therapeutics Submits New Drug Application for Relacorilant to Treat Cushing's Syndrome
• Corcept Therapeutics has submitted a New Drug Application to the FDA for relacorilant, a selective cortisol modulator designed to treat patients with endogenous hypercortisolism (Cushing's syndrome).
• The application is supported by positive results from multiple clinical trials, including the pivotal GRACE trial, Phase 3 GRADIENT study, and long-term extension studies, demonstrating improvements in various symptoms with an acceptable safety profile.
• Relacorilant showed no instances of serious adverse events common in current treatments, such as drug-induced adrenal insufficiency, hypokalemia, or QT prolongation, positioning it as a potential new standard of care.
FDA Approves Subcutaneous Opdivo Qvantig for Multiple Solid Tumors
• The FDA approved Opdivo Qvantig, a subcutaneous injection of nivolumab, for adult solid tumors previously approved for intravenous Opdivo.
• CheckMate-67T trial data showed non-inferior pharmacokinetic exposures, similar efficacy, and comparable safety to intravenous Opdivo.
• Opdivo Qvantig offers a faster 3-5 minute administration time, providing convenience and flexibility for patients.
• The approval helps Bristol Myers Squibb maintain market share amid patent expirations and increasing biosimilar competition.
FDA Grants Orphan Drug Designation to EXG110 Gene Therapy for Fabry Disease
• The FDA has granted orphan drug designation to EXG110, a novel gene therapy developed by Exegenesis Bio for Fabry disease.
• EXG110 is designed as a one-time treatment to deliver a genetic payload directly to liver and heart cells, potentially improving efficacy and safety.
• A clinical trial in China has already dosed its first patient, and plans are underway to launch a US-based clinical trial for EXG110.
• Fabry disease, a rare lysosomal disorder, leads to lipid accumulation and can cause renal failure, cardiac disease, and strokes, highlighting the need for new treatments.
Cytokinetics' Aficamten Advances in Regulatory Review for Obstructive Hypertrophic Cardiomyopathy
• The EMA has validated Cytokinetics' Marketing Authorization Application (MAA) for aficamten, a cardiac myosin inhibitor, for treating obstructive hypertrophic cardiomyopathy (HCM).
• The FDA has accepted the New Drug Application (NDA) for aficamten with a PDUFA target action date of September 26, 2025, and no advisory committee meeting is planned.
• Aficamten significantly improved exercise capacity and clinical outcomes in the SEQUOIA-HCM Phase 3 trial, supporting regulatory submissions in the U.S., Europe, and China.
Advancements in Clinical Trials for Fabry Disease, C. difficile Infections, and Mydriasis
• Clinical trials are actively progressing for Fabry Disease, with companies like Idorsia and Sanofi Genzyme developing novel therapies.
• Emerging treatments like Venglustat and Pegunigalsidase alfa are expected to significantly impact the Fabry Disease market in the coming years.
• Research and development efforts are also focused on Clostridium Difficile Infections, with Vedanta Biosciences leading Phase 3 trials for VE303.
• Mydriasis treatments are advancing, with companies like Eyenovia exploring solutions like MYDCOMBI for pupil dilation.
Pompe Disease Pipeline Shows Promise with Novel Therapies in Development
• The Pompe disease pipeline is robust, featuring over 20 drugs in development across 15+ companies, targeting novel treatment approaches.
• GeneCradle Therapeutics' GC301, a gene therapy, is in Phase I/II trials, showing potential for enzyme replacement therapy discontinuation and motor ability improvement.
• Maze Therapeutics' MZE001, an oral glycogen synthase inhibitor, is in Phase I, aiming to reduce glycogen buildup in Pompe disease patients.
• Key players like Amicus Therapeutics and Spark Therapeutics are advancing therapies such as Cipaglucosidase alfa and SPK-3006 through clinical trials.
Bristol Myers Squibb Receives Positive CHMP Opinions for Opdivo, Yervoy, and Breyanzi
• The CHMP recommended Opdivo plus Yervoy for first-line treatment of unresectable or advanced hepatocellular carcinoma (HCC) in adults, based on the CheckMate -9DW trial.
• Breyanzi received a positive CHMP opinion for relapsed or refractory follicular lymphoma after two or more prior systemic therapies, showing a 97.1% response rate in the TRANSCEND FL study.
• Opdivo plus Yervoy also received a positive CHMP opinion for first-line treatment of MSI-H or dMMR metastatic colorectal cancer, demonstrating a 79% reduction in disease progression or death risk.
Sangamo Therapeutics' Fabry Disease Candidate ST-920 Gains Accelerated Approval Pathway
• Sangamo Therapeutics' shares surged after the FDA agreed to a regulatory pathway for accelerated approval of isaralgagene civaparvovec (ST-920) for Fabry disease.
• The FDA will consider data from the Phase I/II STAAR trial, using the rate of decline in eGFR at 52 weeks as the primary basis for approval.
• Sangamo plans to submit a BLA in the second half of 2025, three years ahead of previous estimates, potentially bringing the treatment to patients sooner.
• Septerna, focusing on GPCR therapies, raised $288 million in an upsized IPO, highlighting renewed interest in biotech IPOs.
Advancements in Pompe Disease Treatment: A Pipeline Review
• Pompe disease, a rare genetic disorder, is seeing advancements with over 20 therapies in the pipeline.
• Key players like Amicus Therapeutics and Spark Therapeutics are developing novel treatments, including gene therapies and enzyme replacements.
• Clinical trials are underway, evaluating the safety and efficacy of drugs like Cipaglucosidase alfa and SPK-3006.
• These emerging therapies target various mechanisms, such as alpha-glucosidase replacement and glycogen synthase kinase modulation.
Vutrisiran Shows Promise in ATTR-CM Treatment: Alnylam's HELIOS-B Data Highlights Cardiac Benefits
• New data from the HELIOS-B study reveals vutrisiran significantly improves cardiac structure and function in ATTR-CM patients over 30 months compared to placebo.
• Vutrisiran demonstrates stability in cardiac biomarkers NT-proBNP and Troponin-I, indicating a potential disease-modifying effect, particularly when administered early.
• Alnylam has submitted regulatory applications to the FDA and EMA for vutrisiran, aiming to establish it as a first-line therapy for ATTR amyloidosis with cardiomyopathy.
• The HELIOS-B trial's results, published in NEJM, underscore vutrisiran's potential to reduce mortality and cardiovascular events in ATTR-CM patients.
© Copyright 2025. All Rights Reserved by MedPath