Amicus Therapeutics

DelveInsight's 'Pompe Disease Pipeline Insights, 2024' covers 15+ companies and 20+ pipeline drugs, including clinical and nonclinical stage products, with insights on product type, stage, route of administration, and molecule type. Key companies include Spark Therapeutics, Amicus Therapeutics, and Novartis AG. Report highlights recent clinical trial results and emerging therapies.

DelveInsight's 'Fabry Disease Pipeline Insight, 2024' report details 18+ companies developing 18+ therapies, including Venglustat, Pegunigalsidase alfa, and 4D 310, with insights on mechanism of action, route of administration, and clinical trials. Key companies include Idorsia Pharmaceuticals, Protalix, Sanofi Genzyme, and Sangamo Therapeutics. The report covers pre-clinical to marketed phases, focusing on commercial and clinical assessments.

The 2025 Patients as Partners meeting focuses on patient-centric drug development, advocacy integration, and clinical trial efficiencies, featuring keynotes by Jayne Gershkowitz, Dr. Ify Osunkwo, Dr. Justin Hopkin, and Anthony Yanni. Topics include diversity, patient engagement measures, AI, digital tech, and regulatory updates.

BioNJ launches 'Time Off for Clinical Trials' initiative, encouraging member companies to offer paid time off for employees to participate in clinical trials. Amicus Therapeutics, Bristol Myers Squibb, Genmab, PsychoGenics, PTC Therapeutics, and Sanofi are among the initial companies committed to implementing this policy.

BioNJ launches 'Time Off for Clinical Trials' initiative, encouraging member companies to offer paid time off for employees to participate in clinical trials. Amicus Therapeutics, Bristol Myers Squibb, Genmab, PsychoGenics, PTC Therapeutics, and Sanofi are among the first to commit. The initiative aims to remove barriers to participation in clinical trials, particularly for hourly workers and underrepresented populations.

Amicus Therapeutics and Teva Pharmaceuticals have reached an agreement allowing Teva to sell a generic version of Galafold (migalastat) for Fabry disease in the U.S. starting Jan. 30, 2037, pending FDA approval. The settlement resolves a legal dispute that began in 2020, and Amicus remains engaged in a separate dispute with Aurobindo Pharma. Galafold, a chaperone therapy, stabilizes the defective Gal A enzyme to slow Fabry disease progression, and is priced at $315,000 per year.

Blue Trust Inc. increased its holdings in Amicus Therapeutics by 1,705.4% in Q3, owning 2,997 shares worth $30,000. Other investors also adjusted their stakes. FOLD shares opened at $11.47, with a 50-day moving average of $11.15 and a 52-week range of $9.02-$14.57. Amicus Therapeutics reported $126.67 million in revenue for Q2, up 34% YoY. Analysts predict -0.06 EPS for the current fiscal year. CEO Bradley L. Campbell sold 7,500 shares at $10.60 each, reducing his position by 0.00%. Amicus Therapeutics focuses on rare disease treatments.

Canadian approach to supply chain issues reduced drug shortages by 40% compared to the US, Biden hosts AI-biotech summit, FTC appeals noncompete ban ruling, US drugmakers seek alternatives to Chinese supply chains, generic drugmakers settle price-fixing charges, schizophrenia drug shows mixed results, CRISPR gene editing advances, debate on race in clinical algorithms, and Abbott and Reckitt win baby formula trial.

Sangamo Therapeutics' shares surged 69% after FDA alignment on an Accelerated Approval pathway for its Fabry disease gene therapy, isaralgagene civaparvovec (ST-920), using data from its Phase I/II STAAR trial. The company plans to submit a BLA in the second half of 2025, three years ahead of previous estimates.