What We’re Reading: Rare Disease Drug Approved; Congress and PBMs; FDA Panel Splits on RSV Shot

The FDA approved the first drug for the rare disease Friedreich’s ataxia; House Republicans seek information from pharmacy benefit managers (PBMs); FDA panel narrowly supports respiratory syncytial virus (RSV) vaccine for older adults.

Rare Disease Drug for Friedreich’s Ataxia Approved

The FDA approved Reata Pharmaceuticals’ rare disease drug, omaveloxolone (Skyclarys), the first approved treatment for the rare genetic disorder Friedreich’s ataxia, which causes progressive nervous system damage and can lead to early death. The drug is the firm’s first approved therapy, according to Reuters.

Congress Drug-Price Investigation Focuses on Pharmacy Benefit Managers

An investigation into companies that manage drug benefits has been initiated by House Republicans, increasing the attention placed on pharmacy benefit managers (PBMs) who play a role in medication costs, reported The Wall Street Journal. The House Oversight and Accountability Committee said that it sent letters to the largest PBMs, consisting of CVS Health Corp.’s CVS Caremark, Cigna Group’s Express Scripts, and UnitedHealth Group Inc.’s OptumRx, looking for documents about their negotiated drug-price rebates and fees they charge.

FDA Panel Just Barely Backs Pfizer RSV Vaccine

An FDA advisory panel narrowly supported an experimental Pfizer vaccine for respiratory syncytial virus (RSV), voting 7-4 on 2 separate questions regarding Pfizer’s safety and efficacy data for people aged 60 and older, reported The Associated Press. One of the panelists abstained from voting. The recommendation is not binding, and the FDA will make a decision on the vaccine in the coming months. The favorable vote came in spite of concerns about possible rare reactions and questions about how the vaccine affected people who have the greatest risks from RSV.

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