Drugs

Reata Pharmaceuticals’ Friedreich’s Ataxia Drug Approved

FDA has approved a Reata Pharmaceuticals NDA for Skyclarys (omaveloxolone) as the first treatment for Friedreich’s ataxia, a rare degenerative disease that damages the nervous system. The disease, which is characterized by impaired coordination and walking, causes progressive damage to the spinal cord, peripheral nerves, and the brain, resulting in uncoordinated muscle movement, changes in speech and swallowing, and a shortened lifespan, according to an FDA release. It typically develops in children and teenagers and worsens over time.

The approval was based on data from a 48-week randomized, placebo-controlled, and double-blind study. “The primary objective was to evaluate the change in the modified Friedreich’s Ataxia Rating Scale (mFARS) score compared to placebo at week 48,” the agency says. “The mFARS is a clinical assessment that measures disease progression, namely swallowing and speech (bulbar), upper limb coordination, lower limb coordination, and upright stability. Individuals receiving Skyclarys performed better on the mFARS than people receiving placebo. In a post hoc analysis, individuals who continued treatment with Skyclarys in an open-label extension for up to three years performed better on the mFARS compared to a matched set of untreated patients from a natural history study.”

FDA says Skyclarys’ most common side effects were increases in alanine transaminase and aspartate aminotransferase, which can be signs of liver damage. Headache, nausea, abdominal pain, fatigue, diarrhea and musculoskeletal pain were also common in the study.

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

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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