Six weeks after a reduction in force slashed the Food and Drug Administration’s workforce, signs are emerging that its drug approval process may change. That’s a concern across the pharmaceutical industry. After all, the agency faces live fire every time an approved medication leads to severe side effects or deaths.
On Tuesday, FDA Commissioner Marty Makary unveiled a new program to expedite the review of drugs that the administration deems important for public health. The program grants “vouchers” to companies whose products align with FDA goals and thereby deserve the fastest reviews. The vouchers don’t alter the scientific requirements for approval of new medicines, but they do allow the agency to waive its requirement that randomized clinical trials be conducted before a drug is given a green light for sale.
As for 2024, C&EN’s tabulation includes only the first approvals under the new scheme. The top picks include Bristol Myers Squibb’s antipsychotic KarXT (bresoximab) for schizophrenia and small biotech Madrigal Pharmaceuticals’ resmetirom, which is targeted at non-alcoholic fatty liver disease, an increasingly prevalent condition that can lead to liver failure and cancer. Evaluate, which estimates sales by 2028, projects $2.8 billion in potential revenues for the two treatments.
The runner-up is a muscle-wasting treatment from Basilea Pharmaceutics, a company that’s hoping its Zolgensma can help patients with amyotrophic lateral sclerosis. Several of the year’s other approvals are antibiotics, including Allecra Therapeutics’ Exblifep (cefepime and enmetazobactam) and Basilea’s Zevtera (ceftobiprole medocaril sodium).