After Recent Leadership Departures, Is the FDA Returning to Form?

Some experts say the FDA's recent change of heart on a handful of drugs for rare diseases suggests a return to regular order after a tumultuous year, while others argue the flip-flopping signals more chaos.

During the tenure of former FDA Commissioner Marty Makary, MD, MPH, and the agency's top vaccine official Vinay Prasad, MD, MPH, several rare disease drugs were rejected or sponsors were asked to complete additional studies.

For example, in January, the FDA sent a complete response letter to Atara Biotherapeutics and its partner Pierre Fabre Pharmaceuticals rejecting the T-cell immunotherapy tabelecleucel (Ebvallo) for the treatment of Epstein-Barr virus-positive post-transplant lymphoproliferative disease. The next month, the agency rejected Regenxbio's gene therapy RGX-121 for mucopolysaccharidosis II, also known as Hunter syndrome.

Then in March, the FDA closed the door on an accelerated approval of AMT-130, uniQure's treatment for Huntington's disease, sparking controversy by recommending a sham surgery-controlled study. Finally, in April, the agency panned Replimune's drug candidate for treating advanced melanoma.

Prasad left the agency in March, and Makary resigned in May.