Nov. 29 Quick Takes: Karuna’s schizophrenia therapy gets FDA goal date

Karuna Therapeutics Inc. (NASDAQ:KRTX) said FDA has accepted its NDA for KarXT to treat schizophrenia and issued a PDUFA date of Sept. 26, 2024. The therapy pairs xanomeline, an agonist of muscarinic acetylcholine receptors M1 and M4, with trospium chloride, an antagonist of M2 and M3. It is designed to mitigate xanomeline’s side effects, including sedation, weight gain and extrapyramidal motor effects. KarXT has led to significant reductions in psychosis scores in multiple studies. PureTech Health plc (LSE:PRTC; NASDAQ:PRTC) co-founded Karuna.

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) announced an agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) to sell its Roctavian valoctocogene roxaparvovec gene therapy for severe hemophilia A in Germany for about $900,000 per patient. In the U.S., BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) has priced Roctavian at a wholesale acquisition cost of $2.9 million. The company has committed to provide an outcomes-based warranty to all government and commercial payers in the U.S. that will reimburse up to 100% of the cost if a person does not respond to the treatment...