Infrequent
Applied Therapeutics’ Rare Disease Drug Fails Another Phase 3 Trial After FDA Rejection
Applied Therapeutics; govorestat; rare disease; phase 3 failure; FDA rejection; classic galactosemia; aldose reductase inhibitor; clinical trial conduct; FDA warning letter
BioMarin Acquires Inozyme Pharma for $270M, Gaining Phase 3 Rare Disease Therapy
BioMarin; Inozyme Pharma; acquisition; rare disease; INZ-701; ENPP1 Deficiency; enzyme replacement therapy; Phase 3 clinical trial
FDA Delays Decision on Biohaven’s Troriluzole for Rare Disease, Plans Advisory Committee Review
Biohaven; FDA delay; troriluzole; spinocerebellar ataxia (SCA); rare disease drug; PDUFA extension; advisory committee; neurodegenerative disease
J&J’s $130M gene therapy bet flunks rare retinal disease trial
Johnson & Johnson, gene therapy, bota-vec, X-linked retinitis pigmentosa, Phase 3 failure, vision loss treatment, rare eye disease, $130 million investment
FDA chief Makary talks expediting drug approvals, RIFs and the abortion pill
United States Food and Drug Administration, Califf, Unspecified Abortion, Contraceptives, Oral, Accelerated, Artificial Intelligence, RIFs, Infrequent, Agencies
Abeona’s $3.1M gene therapy wins FDA nod for rare skin disease
United States Food and Drug Administration, United States Food and Drug Administration, Infrequent, ZEVASKYN, Abeona, gene therapy, Dermatologic disorders, Hallopeau-Siemens Disease, Approved, Abeona ‘s
Merck KGaA closes in on SpringWorks deal at $3.5B price point
SpringWorks, Acquisition (action), KGaA, Infrequent, Gomekli, deal, Merck
Glycomine raises $115M to see if it can treat rare glycosylation disorder
Infrequent, GLM101, Glycomine, Congenital disorder of glycosylation type 1A
Arbor sails into rocky gene editing waters with $74M for rare liver disease therapy
Gene editing, CRISPR, Rare liver disease, Primary hyperoxaluria type 1 (PH1), Funding
FDA Approves Ono Pharmaceutical’s Romvimza for Rare Joint Tumor
Romvimza, tenosynovial giant cell tumor (TGCT), FDA approval, Ono Pharmaceutical, Deciphera acquisition, kinase inhibitor, rare cancer treatment