Denali Therapeutics’ Avlayah received FDA approval for treating Hunter syndrome, a rare neurological disorder. The biologic medicine uses Denali’s proprietary drug delivery technology to cross the protective blood-brain barrier.
Leucovorin is now approved for cerebral folate deficiency months after FDA Commissioner Marty Makary claimed the decades-old generic drug had promise for treating autism. The FDA’s review was based on published literature and real-world evidence.
Zelis highlights how employers, payers, and consumers are integrating digital tools to manage healthcare costs.
Povetacicept, a Vertex Pharmaceuticals immunology drug designed to block two targets, met the main goal of a Phase 3 clinical trial in the rare kidney disease immunoglobulin A nephropathy. The fusion protein came from Vertex’s acquisition of Alpine Immune Sciences, and analysts say the drug has best-in-class potential.
GSK licensed to Alfasigma global rights to linerixibat, a drug developed to treat the rare liver disease primary biliary cholangitis (PBC). The move follows Alfasigma’s 2025 voluntary market withdrawal of Ocaliva, a PBC drug that had sparked safety concerns from U.S. and European regulators.
The Series B extension brings Atavistik Bio’s Series B financing to $160 million. The startup’s lead program is in development for hereditary hemorrhagic telangiectasia (HHT), an inherited bleeding disorder.
Ampreloxetine’s Phase 3 failure in multiple system atrophy is the third disappointing clinical trial outcome for the Theravance Biopharma drug. The biotech will now wind down all R&D and explore options that could include the sale of the company.
MedCity News was at the Vive conference and spoke with executives who shared their insights for the healthcare industry.
Atrium spun out of Avidity Biosciences, which Novartis acquired in a $12 billion deal. The Atrium pipeline is led by two RNA therapies with a path to the clinic for rare inherited cardiological disorders that have no FDA-approved drugs.
It's not just about awareness but also a call to action for patients, families, employers, payers and policymakers to modernize the systems that shape access to care. It must focus on connecting innovation with the infrastructure that governs approval, coverage and affordability.
The FDA asked for more data to determine whether Disc Medicine’s bitopertin is benefiting patients with erythropoietic protoporphyria, a rare blood disorder. Analysts say it’s a surprising delay considering bitopertin is one of the first drugs in a new FDA pilot program that speeds up regulatory review of medicines deemed critical for national security or public health.
PTC Therapeutics said it cannot resolve differences it has with the FDA in interpreting clinical trial data for Translarna, a drug it developed for Duchenne muscular dystrophy. The rare disease drug developer’s decision to withdraw Translarna’s FDA submission comes nearly a year after a negative European regulatory decision for the drug.
Artera President Tom McIntyre talks about the practical application of AI in healthcare.
Infigratinib achieved statistically significant improvement in growth rate and body composition in a Phase 3 clinical trial that tested the BridgeBio Pharma drug in achondroplasia, the most common form of dwarfism. Regulatory submissions are planned for later this year.
Brepocitinib is on track for pivotal testing after meeting the goals of a proof-of-concept study in cutaneous sarcoidosis, a rare inflammatory skin disorder with no FDA-approved therapies. Roivant Sciences subsidiary Priovant Therapeutics licensed the dual JAK1 and TYK2 inhibitor from Pfizer.
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.
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.