The Digital Medicine Society (DiME) launched a new set of FDA-aligned digital measures to standardize how outcomes are tracked in pediatric rare disease trials. The framework aims to make trials faster and more efficient, giving researchers and drugmakers a practical roadmap for developing new therapies for children who often have no effective treatment options.
Tests of two Regenxbio gene therapies have been placed under an FDA clinical hold after a patient in one of the studies developed cancer. Our recap of recent regulatory news also includes one clinical hold removed, several complete response letters, and drug approvals in the U.S. and Europe.
How can AI benefit and improve patient outcome in the emergency department? Watch the latest episode to find out.
Zenas Biopharma’s obexelimab achieved a statistically significant and clinically meaningful reduction in signs and symptoms of immunoglobulin G4-related disease, but not to the extent shown by Amgen’s Uplizna in its pivotal study. Still, Zenas points to features of its drug that could make it competitive as a maintenance therapy for the rare autoimmune disorder.
Omeros’s complement system drug Yartemlea is now FDA approved for treating a severe and potentially fatal complication of hematopoietic stem cell transplants. Our recap of other regulatory actions includes drug approvals in cancer, rare disease, and metabolic disorders.
BioMarin Pharmaceutical’s acquisition of Amicus Therapeutics is a marriage of two rare disease biotech companies. Amicus’s two commercialized products, for the enzyme deficiencies Fabry disease and Pompe disease, are each projected to become blockbuster sellers.
Waskyra is the first FDA-approved gene therapy for the rare disease Wiskott-Aldrich syndrome and the first such approved product from a non-profit applicant, Fondazione Telethon. Data from Waskyra’s pivotal studies were presented this week during the annual meeting of the American Society of Hematology.
Greenway Health blends smart technology with human expertise to elevate revenue cycle.
Protagonist Therapeutics’ drug rusfertide reduced the need for phlebotomies as a way to treat the rare blood cancer polycythemia vera. Longer-term Phase 3 data for the Takeda Pharmaceutical-partnered peptide drug were presented during the annual meeting of the American Society of Hematology.
Pharvaris’s deucrictibant met the main and secondary goals of its Phase 3 test as an on-demand treatment for swelling attacks caused by the rare disease hereditary angioedema. Pharvaris’s capsule could compete against Kalvista Pharmaceuticals’ Ekterly, an HAE pill approved by the FDA over the summer.
Regeneron Pharmaceuticals will share in development of Tessera Therapeutics’ TSRA-196, a Phase 1-ready gene-editing therapy for alpha-1 antitrypsin deficiency (AATD). Other companies developing genetic medicines for this rare disease include Beam Therapeutics, Wave Life Sciences, Korro Bio, and AIRNA.
Protego Biopharma’s lead program is a potential treatment for the rare disease light chain amyloidosis. The startup says the novel mechanism of its oral small molecule should have better outcomes than antibodies from AstraZeneca and Prothena that failed their respective pivotal studies in the disorder earlier this year.
In a recent interview, Esperion president and CEO, Sheldon Koenig talks about the reality of statin intolerance and his company’s expansion into new diseases
Arrowhead Pharmaceuticals’ RNAi drug plozasiran, brand name Redemplo, is now the second FDA-approved therapy for familial chylomicronemia syndrome, an inherited lipid disorder. Beyond its claims of safety and dosing advantages, Arrowhead set a dramatically lower price for its first commercial product, which will compete against an Ionis Pharmaceuticals drug.
Eli Lilly is paying $75 million up front for global rights to AAV-AIPL1, a gene therapy on track for U.S. and European regulatory submissions in the rare, inherited eye disorder Leber congenital amaurosis 4, or LCA4. The deal brings to Lilly another late-stage gene therapy for the eyes following the pharma giant’s October acquisition of Adverum Biotechnologies and its lead program for wet AMD.
The six new drugs awarded FDA Commissioner’s National Priority Vouchers brings to 15 the total number of therapies selected for the new pilot program. Under this program, the FDA says the standard 10- to 12-month review time could be shaved down to “within months.”
The FDA has approved UCB’s Kygevvi for treating children and adults with thymidine kinase 2 deficiency, an inherited mitochondrial disorder. It’s the drugmaker’s first ultra-rare disease product.
Sarepta Therapeutics attributed the Phase 3 failure to the Covid-19 pandemic, during which many patients missed multiple doses of its Duchenne muscular dystrophy therapies. The company plans to discuss with the FDA traditional regulatory approvals based on the totality of data that includes real world evidence from the years these drugs have been commercially available under accelerated approvals.