The news: Avidity Biosciences struck a deal with the FDA for accelerated approval of a potential treatment for a rare type of muscular dystrophy.

Zooming in: Avidity is beginning a phase 3 study for the drug candidate that targets the underlying cause of faciopulohumeral muscular dystrophy (FSHD). The muscle-wasting disease affects 45,000 to 87,000 people in the US and Europe but has no approved treatments.

The accelerated approval pathway allows the FDA to fast-track a drug that treats serious conditions with unmet need based on achieving a specific endpoint versus proving clinical benefit. The FDA gave Avidity detailed directions on what’s necessary to hit its mark, per the company.

Zooming out: Biotech and pharma companies expected the focus on expediting accelerated approval to carry over from Trump’s first term when he took office again. However, mass FDA layoffs, delayed drug approval dates, and sparse communications left accelerated approval reform on the back burner.

• Accelerated approval dropped an average of 2 years from oncology drugs’ time to market, from 4.3 years to 2.3 years, according to a recent study in the Journal of Cancer Policy. 
• Drugs that had to be withdrawn dropped even more dramatically, from 9.5 years to 3.2 years.

Why it matters: Accelerated drug approval may be getting renewed attention via HHS’ recent call for deregulation input. 

At a cell and therapy roundtable last week, HHS Secretary Robert F. Kennedy Jr and health agency heads, including FDA chief Marty Makary and Centers for Biologics Evaluation and Research (CBER) director Vinay Prasad, asked scientists and experts for a list of what regulations they want to remove.

Trump health officials pointed to the example of baby KJ Muldoon, who received the first custom gene therapy created specifically for his rare genetic mutation in only a few months, as what’s possible with more relaxed regulations, per Fierce Biotech. In April, FDA’s Makary said in a Fox News interview that the agency is exploring conditional accelerated pathways for rare disease drugs. 

Our take: The Avidity announcement and federal health agency enthusiasm are positive signs for biotech and pharma companies looking for accelerated approval for rare disease candidates. Federal authorities and drugmakers will need to balance speed-to-market with rigorous science to ensure physician and consumer confidence.

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