Drugs

FDA OKs Higher Dose Regimen for Spinraza

FDA has approved a higher-dose regimen of Biogen’s Spinraza (nusinersen) for treating spinal muscular atrophy, giving patients and clinicians a new dosing option aimed at improving outcomes in the rare neuromuscular disorder. The decision is based on data from the Phase 2/3 DEVOTE study and expands on more than a decade of clinical experience with the drug’s original 12 mg regimen, the company says.

The newly approved regimen includes higher loading and maintenance doses — two 50 mg doses administered two weeks apart for new patients, followed by 28 mg maintenance doses every four months. Patients already on the standard regimen can transition after a single high-dose loading phase, according to the company.

In the DEVOTE trial, treatment-naive infants receiving the higher-dose regimen showed statistically significant improvements in motor function compared with a matched untreated control group. The primary endpoint, measured by the CHOP-INTEND scale, demonstrated a mean improvement of more than 15 points versus a decline in the control group, according to Biogen.

Spinraza, an antisense oligonucleotide therapy delivered intrathecally, was first approved in 2016 and marked a major turning point for SMA, a genetic disease that leads to progressive muscle weakness and, in severe cases, early mortality.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

Read more

Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome