Trending News Today: FDA Rejects Accelerated Approval for Duchenne Muscular Dystrophy Drug

Top news of the day from across the health care landscape.

More women should be assessed for harmful mutations in the BRCA1 and BRCA2 genes that can increase the risk of certain cancers, according to CNN. The US Preventative Task Force has updated its previous 2013 recommendation for cancer risk assessment to include women who personally have been diagnosed with breast, ovarian, or tubal cancers in the past but completed treatment and are considered "cancer free," and those who have certain ancestries associated with BRCA1 or BRCA2 mutations, such as being of Ashkenazi Jewish descent. The updated recommendation notes that for women who are recommended for screening for BRCA1 or BRCA2 mutations, that genetic risk assessment and BRCA mutation testing is a multi-step process, which typically begins with assessing risk at a visit with a primary care doctor.

Patients with allergic asthma could benefit from allergy immunotherapy (AIT) to prevent the condition from progressing, according to MD Magazine. A team of investigators recently completed a population-based cohort study involving approximately 1.7 million patients using health care data derived between 2005 and 2014 from statutory health insurance in Germany. AIT exposure was associated with a significantly decreased likelihood of asthma progression from global initiative for asthma (GINA) step 1 to GINA step 3 (HR, 0.87; 95% CI, .80‐.95) and GINA step 3 to GINA step 4 (HR, .66; 95% CI, .60‐.74). GINA medication for step 2 and step 5 were rarely prescribed.

In a highly anticipated decision, the FDA on Monday rejected accelerated approval for Sarepta Therapeutics’ second Duchenne muscular dystrophy (DMD) drug, according to the American Journal of Managed Care. Golodirsen, or Vyondys 53, is an investigational injection for the treatment of DMD—a rare muscle-wasting disease typically seen in males between 1 and 6 years of age—in individuals with a confirmed mutation amenable to exon 53 skipping. In the company’s statement, Sarepta said the FDA’s complete response letter cites concerns over risks of infections related to intravenous infusion ports, as well as renal toxicity seen in preclinical models of the drug and observed following administration of other antisense oligonucleotides.