The FDA’s Accelerated Approval Program provides an avenue to encourage pharmaceutical manufacturers to develop urgently needed new medications and to make them available to patients as quickly as possible. The focus of this program is to address serious medical conditions for which there are limited or no treatments available. The drugs and vaccines considered for the Accelerated Approval Program include those developed to treat infectious diseases, cancer, and non-malignant blood and neurological disorders. Rather than requiring lengthy clinical trials for initial approval, accelerated approval is granted based on a surrogate endpoint, which is a marker that predicts clinical benefits. This can include physical signs, laboratory measurements, radiographic images or other measures. Although accelerated approval helps to shorten the time required before a medication is available to treat patients, the approval is only temporary. Pharmaceutical companies must conduct larger clinical trials following accelerated approval of the medication and provide the FDA with data from these trials to confirm the medication is effective. If these confirmatory trials are successful, the FDA will grant traditional approval for the drug or vaccine. If these confirmatory trials have unsatisfactory results and do not show clinical benefit, the FDA may remove the drug from the market.
On November 22, 2022, the pharmaceutical and biotechnology company GSK announced it would be withdrawing Blenrep (belantamab mafodotin-blmf) from the U.S. market. Blenrep received accelerated approval from the FDA in 2020 after presenting tumor shrinkage data in the treatment of multiple myeloma from two early trials. However, its confirmatory study, DREAMM-3, failed to meet its expected effectiveness endpoints and led to the withdrawal of the medication from the market. Several other treatments have had a similar outcome this year, including Ukoniq®, Marqibo® and Farydak®. Other medications, such as Tecentriq®, Zydelig® and Keytruda®, have remained on the market but have lost approval for some of their indications because confirmatory trials failed to show benefit for these conditions. Blenrep has two other studies in progress for use in combination with novel or standard medications to treat multiple myeloma, but GSK will need to submit a new biologics license application for FDA approval if the combinations are found to be effective.