DUBLIN – Shares in Santhera Pharmaceuticals AG surged by 67% June 1 on news that the high-dose arm of a phase IIb pivotal trial of vamorolone hit the primary endpoint of an improvement vs. placebo in the time-to-stand velocity attained by ambulatory boys with Duchenne muscular dystrophy (DMD). The Pratteln, Switzerland-based firm aims to file an NDA in the first quarter of next year, for which it will seek a priority review from the FDA, and a marketing authorization application in Europe in the second quarter of 2022.

Alkermes’s first oral anti-psychotic therapy gets the FDA’s approval

Alkermes plc worked its way through a complete response letter issued in late 2020 as the FDA has approved Lybalvi (olanzapine and samidorphan) for treating adults with schizophrenia and bipolar I disorder. The once-daily, oral antipsychotic treatment, the company’s first, is for maintenance monotherapy or acute treatment of manic or mixed episodes and as a monotherapy or adjunct to lithium or valproate. The therapy had a June 1 PDUFA date. In November, regulators reviewed manufacturing records and said the Dublin-based firm needed to supply documents related to making the tablet’s coating at the Wilmington, Ohio, facility. No clinical or nonclinical concerns were raised at the time, the firm said.

Lumakras approved in NSCLC, Amgen talks about pricing and potential

Amgen Inc. held a conference call to discuss the FDA clearance last week of Lumakras (sotorasib), the first targeted therapy for adults with KRAS G12C-mutated locally advanced or metastatic non-small-cell lung cancer, as determined by an FDA-approved test. The company priced the compound at $17,900 per month, somewhat lower than expected by analysts such as Piper Sandler’s Christopher Raymond. On the call, Amgen officials answered questions about the launch of Lumakras and its prospects as a combo therapy.

Sanofi halts pivotal test of venglustat in ADPKD for futility

Interim findings that Sanofi SA's venglustat is unlikely to provide a meaningful benefit to people with autosomal dominant polycystic kidney disease (ADPKD) have caused it to halt a phase II/III study of the candidate. Testing continues in other rare diseases, but the exit could be a boon for Otsuka Pharmaceutical Co. Ltd.'s Jynarque (tolvaptan), its generic competitors and ascendant midstage novel ADPKD programs still underway.

Safety signal halts work on AB Science’s masitinib

Trading in shares in AB Science SA was suspended Tuesday after a safety signal prompted a voluntary hold on two late-stage clinical studies of its masitinib, in mastocytosis and amyotrophic lateral sclerosis, respectively. The Euronext-listed company also put a phase II trial in COVID-19 on hold after it identified a potential risk of ischemic heart disease with masitinib, following discussions with regulators including France’s ANSM.

Diverse cohort expands diabetes genomics

Data on the prevalence of diabetes in the U.S. show that non-Hispanic white people are least likely to suffer from the disease. Yet to date most genetic studies of the glycemic traits that are used to diagnose and monitor type 2 diabetes and cardiometabolic health have focused on individuals of European ancestry. The same is true in other multifactorial diseases, a reality that undermines moves toward precision medicine and threatens to further exacerbate health disparities. A new study in the May 31, 2021, online issue of Nature Genetics goes some way to address this, in a genome wide association study (GWAS) of 281,416 individuals without diabetes, of whom 30% had non-European ancestry. The improved power and resolution this mix provided led to the identification of 242 loci, of which 99 were novel and had not previously been associated with any of four glycemic traits or with type 2 diabetes. The researchers say a comparable analysis restricted to individuals of European ancestry would have identified only 218 loci.

Vaccine vigilance in a sea of unknowns, as world limps away from COVID-19

Jam-packed with efforts to address COVID-19 variants and next-step booster shots, as well as safety concerns over vaccine co-administrations and the expansion of emergency use authorizations (EUAs) into younger populations, May was another busy pandemic-fighting month for the world, despite higher numbers of those vaccinated and declining death and hospitalization rates. India struggled throughout the month, reaching record cases and deaths that have started to decline. The debate over a proposed World Trade Organization (WTO) intellectual property waiver dominated the political arenas. The anticipated Novavax Inc. vaccine has hit more delays, pushing a potential fourth vaccine for the U.S. back to the third quarter. And Vir Biotechnology Inc.’s sotrovimab gained an EUA for mild to moderate disease in high-risk individuals 12 and older.

HHS restores Unapproved Drugs Initiative

The Biden administration is reviving the U.S. FDA’s Unapproved Drugs Initiative, which was terminated late last year by the Trump administration. Lawmakers frequently have called out the program in congressional hearings on drug prices as one more example of biopharma shenanigans, noting that some companies were using it to obtain de facto exclusivities for drugs that had been available for decades and then increase the prices of those drugs. In reversing the termination of the initiative, the Department of Health and Human Services (HHS) cited legal and factual inaccuracies in the Trump notice ending the program and committed the FDA to issuing guidance with updates on its enforcement priorities for unapproved drugs that remain grandfathered on the U.S. market.

Holiday notice

BioWorld's offices were closed in observance of Memorial Day in the U.S. No issue was published Monday, May 31.

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