With two drugs cleared by the U.S. FDA for Niemann-Pick disease type C (NPC) in less than seven days, Wall Street was pondering the differences between the compounds, given what’s known so far about each. Most recently, the FDA approved Intrabio Inc.’s Aqneursa (levacetylleucine) on its PDUFA date for the treatment of neurological manifestations of NPC in adults and pediatric patients weighing at least 15 kg, making Aqneursa the only approved stand-alone therapy indicated for NPC. On Sept. 20, Zevra Therapeutics Inc. won FDA clearance for Miplyffa (arimoclomol) as the first treatment for NPC.
The FDA has cleared an IND application for a phase I/II study of KSQ-004EX, KSQ Therapeutics Inc.’s second engineered tumor-infiltrating lymphocyte (eTIL) program. KSQ-004EX consists of TIL in which the genes encoding SOCS1 and Regnase-1 are inactivated using CRISPR/Cas9 gene editing.
Arrowhead Pharmaceuticals Inc. has filed for regulatory clearance to initiate a phase I/IIa trial of ARO-INHBE, the company’s investigational RNA interference (RNAi) therapeutic being developed for obesity.
The U.S. FDA’s Oncologic Drugs Advisory Committee (ODAC) on Sept. 26 will take up a controversy that’s hardly new: whether approval of immune checkpoint inhibitor drugs should be restricted in accordance with PD-L1 expression.
While in the hot seat at a Sept. 24 U.S. Senate Health, Education, Labor and Pensions (HELP) Committee hearing, Novo Nordisk A/S President and CEO Lars Fruergaard Jørgensen said he would sit down with Sen. Bernie Sanders (I-Vt.) and the three largest pharmacy benefit managers to discuss lowering the list prices for the company’s popular diabetes and weight-loss drugs, Ozempic and Wegovy.
Both the biotech industry and Hong Kong have become strategic points for China as the People’s Republic of China works to lay a biotech “belt and road” through Asia to expand global influence.
The U.S. Court of Appeals for the Federal Circuit reined in a district court that invalidated three claims in an Astellas Pharma Inc. patent protecting bladder drug Myrbetriq (mirabegron) based on a issue that was never argued.
The U.K. National Institute for Health and Care Excellence reported that mental health professionals experienced a dramatic increase in referrals for anxiety and depression over the past decade but noted that digital therapies might help manage the caseload, representing a significant opportunity for developers of these products.
Michelle Tarver, the acting director of the U.S. FDA’s device center appeared on a Sept. 24 webinar to discuss her priorities for the agency going forward, one of which is global health equity.
Chengdian (Suzhou) Biopharmaceutical Co. Ltd. has received IND clearance from the FDA, allowing it to initiate a first-in-human phase I trial of CD-001.
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