Rare Diseases
Pharvaris’s oral bradykinin B2 antagonist hit all endpoints in a Phase III study in hereditary angioedema attacks, meaning the drug could be competing soon with Kalvista’s Ekterly.
The pivotal Phase III trial met the primary and secondary endpoints, showing skeletal and cardiac benefits and supporting deramiocel as a treatment for Duchenne cardiomyopathy.
US FDA approval of the intrathecal formulation of Zolgensma means many more patients with spinal muscular atrophy will be eligible for the gene therapy.
The Phase III portion of the Phase II/III RISE UP study in sickle cell disease met the hemoglobin response primary endpoint, but not the sickle cell pain crisis reduction co-primary endpoint nor a secondary endpoint on reducing fatigue.
Five-year Agamree data shows the novel steroid is a safer and equally effective treatment to standard of care deflazacort or prednisone, the Swiss biotech argues.
After two deaths tied to the gene therapy, Sarepta and the FDA agreed to new labeling for Elevidys, limiting treatment to ambulatory Duchenne muscular dystrophy patients.
Zydus looks to fill up its coffers ahead of the next round of acquisitions, likely in the specialty drugs space, while US FDA approvals for generics to Lynparza and Vumerity and its first approvals in China and Canada point to a healthy pipeline
The German group is looking at ‘single digit billion’ opportunities, chief financial officer Helene von Roeder tells Scrip.
Senior McKinsey executives discuss how biopharma can ace its US launch strategy and first-time launchers can stack the odds in their favor amid complex market dynamics including the MFN pricing push.
The regulatory setback comes as a blow because troriluzole was set to be Biohaven’s sole near-term revenue generator.
UniQure’s plan for a 2026 BLA filing of gene therapy AMT-130 for Huntington’s is uncertain, as the FDA apparently reversed course after indicating it would consider accelerated approval.
Boehringer’s PDE4B inhibitor Jascayd was approved for IPF in China on Oct. 22, marking its second nod globally soon after the US. The German firm discusses with Scrip the opportunities and challenges in this market.
Phase III success for encaleret in autosomal dominant hypocalcemia type 1 gives BridgeBio two pivotal triumphs in three days, following an Oct. 27 readout in a subtype of limb-girdle muscular dystrophy.
Despite a prior agreement with the FDA to seek accelerated approval, with Phase III data showing statistical significance on biomarkers and functional measures, BridgeBio is aiming higher.
The Basel-based company has faith in its existing rare disease commercial platform to successfully market the newly-acquired assets.
The Paris-based group is paying €350m upfront to get hold of Marseille-headquartered ImCheck's acute myeloid leukemia candidate.
Omeros suspended a Phase III program for zaltenibart in May due to financial limitations, but will now get $240m up front from Novo Nordisk for the PNH candidate.
Deal Snapshot: BioCryst is buying Astria in a cash-and-stock deal to get hold of the latter's late-stage injectable hereditary angioedema drug, navenibart, which promises less frequent dosing than Takeda’s market leading injection Takhzyro.
The FDA has extended Denali Therapeutics’ BLA review for its experimental brain-targeted MPS II therapy by three months, pushing the decision date to April 2026.
Leaning on the “totality of the data” despite proof of functional efficacy, some biotechs seek conditional approval for rare disease drugs. The experiences of PTC, Sarepta, Sage and Intercept illustrate the risks of this approach.