EMA’s Committee for Medicinal Products for Human Use (CHMP) has adopted a negative opinion on the MAA related to Zevra Therapeutics Inc.’s arimoclomol for Niemann-Pick disease type C (NPC). Boston-based Zevra said it will ask for a re-examination after CHMP gatekeepers ruled that efficacy has not been sufficiently proven. The U.S. FDA green-lighted the heat shock protein amplifier (used in combination with miglustat) as Miplyffa in September 2024. Through its global expanded access program, Zevra is helping eligible NPC patients get access to arimoclomol and thereby generate additional real-world evidence of its clinical impact. Shares (NASDAQ:ZVRA) were trading at $9.88, down $2.64, or 21%.

CRISPR-focused Scribe goes public via $129M IPO

Continuing the year’s strong IPO activity, Scribe Therapeutics Inc. priced an upsized offering at the high end of its price range, offering 8.6 million shares at $15 apiece for gross proceeds of about $129 million. The company, which is developing CRISPR-based therapies targeting cardiovascular and metabolic diseases, began trading on Nasdaq under the ticker SCTX, with shares getting a warm welcome, trading up 60% at noon.

Medtronic builds out chronic pain continuum with Viaverte, SPR

Medtronic plc’s recent launch of the Viaverte basivertebral nerve ablation system and completion of the acquisition of SPR Therapeutics Inc. broadens its chronic pain offering and strengthens its ability to treat patients across the pain care continuum. “The opportunity to live without pain should be a fundamental right. It shouldn't be a privilege,” Ashwini Sharan, chief medical officer in Medtronic’s neuromodulation business, told BioWorld. The moves come as part of a broader shift in pain management as med-tech companies develop therapies that target the underlying sources of chronic pain, providing alternatives to opioids that have long been associated with risks of addiction, dependence and overdose.

Sanofi scraps plans for amlitelimab filings in atopic dermatitis

Sanofi SA will not seek approval of amlitelimab in atopic dermatitis (AD) as previously planned, stating that the OX40-ligand monoclonal antibody “would not represent a meaningful improvement to the standard of care” for patients with moderate to severe disease. The company said the decision, which marks an about-face as Sanofi had been looking to amlitelimab as a possible successor to its Regeneron Pharmaceuticals Inc.-partnered blockbuster AD drug Dupixent (dupilumab), comes as part of an ongoing assessment of the pipeline. A phase II study of amlitelimab in celiac disease remains ongoing.

APAC roundup: NMPA nods; Lupin’s $1.6M spinoff; CAR T data, more

New drug approvals, financings and deals were seen across the Asia-Pacific biotech sector this week, with China’s National Medical Products Administration (NMPA) granting clearances to Takeda Pharmaceutical Co. Ltd.’s oral orexin receptor 2 agonist oveporexton for narcolepsy, as well as Haisco Pharmaceutical Group’s ciprocopan for paroxysmal nocturnal hemoglobinuria. Lupin Ltd., of Mumbai, India, also spun out two oncology programs to a U.S.-based oncology company, Kaveri Therapeutics Inc., under a $1.6 million deal. And among CAR T-cell therapy companies, Singapore’s Tikva Allocell Pte. Ltd. closed an $8 million series A financing round to support moving lead candidate TAVST-01 into the clinic.

June sets single-month record as global approvals log third-best H1

Global drug approvals reached 261 through the first half (H1) of 2026, the third-highest H1 behind 2021's 288 and 2022's 256, with June's 64 clearances marking the highest single month in BioWorld's records. The U.S. FDA approved 106 drugs through the first six months of the year, with three new molecular entities cleared in June including Viridian Therapeutics Inc.'s Lumvoa (veligrotug) for thyroid eye disease and GSK plc and Spero Therapeutics Inc.'s Utebzi for complicated urinary tract infections.

Also in the news

Accord, Agilent, Alamar, Alligator, Alzpath, Astrazeneca, Atsena, Beone, Caristo, Clinuvel, Eli Lilly, Exubrion, GSK, H. Lundbeck, Haisco, Ibex, Inhibikase, Insight Molecular Diagnostics, Johnson & Johnson, Karl, Koya, Longeveron, Maverix, Modular, Momentis, Nagase Neuphoria, Neuros Medical, Nobias, Nuvectis, Penumbra, Pfizer, Precision, Revolution, Revvity, Scancell, Sentante, Serenity, Serina, Synaptiq, Traferox, Transcripta, United, Veraxa, Verdiva, Viking, Xodus