PTC Therapeutics Inc. reported phase III data in Friedreich’s ataxia (FA) with 15-lipoxygenase inhibitor vatiquinone. The 146-participant study called Move-FA fell short of its primary endpoint of a statistically significant change in modified Friedreich's Ataxia Rating Scale score at 72 weeks in the primary analysis population. On the upside, treatment did demonstrate significant benefit on key disease subscales and secondary endpoints. Shares of South Plainfield, N.J.-based PTC (NASDAQ:PTCT) were trading midday at $47.38, down $11.03, or 19%.
Innoviva’s bacterial infection drug receives FDA approval
A week ahead of its May 29 PDUFA date, the U.S. FDA has approved Xacduro (sulbactam for injection; durlobactam for injection) for treating one of the toughest and most deadly infections, carbapenem-resistant Acinetobacter baumannii, in adults. The combination of beta-lactam antibiotic and broad-spectrum beta-lactamase inhibitor’s approval went to Entasis Therapeutics Inc., which was acquired by Innoviva Inc., of Burlingame, Calif., in July 2022. A. baumannii can become highly resistant to multiple antibacterial drugs and treatment options are limited. Entasis said it expects Xacduro will be available to patients before the end of 2023.
Elevatebio closes $401M series D as Life Edit subsidiary nabs Novo Nordisk deal
In its bid to become, as Chairman and CEO David Hallal said, the “world’s most indispensable cell and gene therapy technology company,” Elevatebio LLC disclosed a $401 million series D round with support from new and existing investors. Among the new investors is R&D partner Novo Nordisk A/S, which concurrently disclosed a potential billion-dollar collaboration with Elevatebio company Life Edit Therapeutics Inc. focused on discovering and developing gene editing therapies for rare genetic disorders and cardiometabolic diseases.
CMS flexes authority in proposing new Medicaid Rx pricing tools
Even as numerous bills that would directly or indirectly impact U.S. prescription drug prices are working their way through Congress, the Centers for Medicare & Medicaid Services (CMS) is flexing its authority in a new proposed rule intended to clamp down on drug prices by providing more transparency in the Medicaid program. The proposed measures would help states and CMS ensure manufacturers appropriately classify their covered outpatient drugs for rebate purposes and, if not, give CMS the ability to take corrective action. Another provision would create additional tools, including a drug price verification survey, to give greater insight into what the most expensive drugs on the market actually cost to manufacture and distribute.
Cansino takes a step closer to COVID-19 mRNA vaccine approval in China
Cansino Biologics Inc. reported positive data in a phase IIb trial evaluating the heterologous mRNA vaccine CS-2034 booster compared to an inactivated vaccine to prevent SARS-CoV-2 infections. The phase IIb trial compared the mRNA vaccine CS-2034 vs. the inactivated BBIBP-CorV by China National Pharmaceutical Group Co. Ltd. as a fourth dose, as well as to evaluate the efficacy against the SARS-CoV-2 omicron (BA.5) variant. Clinical data showed that both CS-2034 and the inactivated vaccine were both well-tolerated as a fourth dose. However, heterologous boosting using CS-2034 induced higher immune responses and protection against symptomatic SARS-CoV-2 omicron infections compared with using the same vaccine.
India ramps up testing requirements after contaminated cough syrup exports
India has revised its policy to make quality tests by government-certified laboratories mandatory for cough syrups to be exported beginning June 1. The decision is the result of numerous safety alerts. Those alerts, from the World Health Organization, warned of substandard cough syrups exported from India, linking them to adverse effects in three countries and deaths of dozens of children in Gambia. The warnings have hit India’s reputation in the pharma sector and its standing as the largest manufacturer of generics in the world. India’s Directorate General of Foreign Trade, which is part of the Ministry of Commerce and Industry, issued a notification on May 23 saying that “cough syrup shall be permitted to be exported subject to the export sample being tested and production of Certificate of Analysis” issued by government-approved laboratories.
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