Two More Accelerated Approval Indications Are Being Pulled

Within the span of one week, Secura Bio, Inc. has revealed that it will withdraw one oncology drug from the U.S. market, as well as an indication for another oncolytic. The FDA had given both accelerated approval. The moves come amid growing scrutiny of that approval pathway, and they mark the ninth and 10th oncology indications and/or drugs taken off the U.S. market since December 2020.

Secura Bio said on Nov. 30 that it will withdraw the new drug application for Farydak (panobinostat). The FDA granted the capsule accelerated approval on Feb. 23, 2015, for the drug in combination with Takeda Pharmaceuticals U.S.A., Inc.’s Velcade (bortezomib) and dexamethasone for the treatment of people with multiple myeloma who have received at least two regimens, including Velcade and an immunomodulatory agent. That indication is the only one the drug has in the U.S. The company said it will continue to market the drug in other areas in which it is approved.

In unveiling the decision, the manufacturer said that “it was not feasible” for it “to complete the required post-approval clinical studies as designed as part of the accelerated approval process.” Novartis Pharmaceuticals Corp. won the approval for the drug, and then Secura Bio purchased the agent in March 2019.

Secura Bio’s decision came just days before a scheduled Dec. 2 meeting of the FDA’s Oncologic Drugs Advisory Committee (ODAC) that was set to review two cancer drugs granted accelerated approval that had not verified clinical benefit: Farydak and Aurobindo Pharma subsidiary Acrotech Biopharma LLC’s Marqibo (vincristine sulfate), which was approved for the treatment of adults with Philadelphia chromosome-negative acute lymphoblastic leukemia in second or greater relapse or whose disease has progressed following at least two anti-leukemia therapies.

Acrotech did not respond by press deadline to an inquiry by AIS Health, a division of MMIT, about whether the company plans to withdraw that indication.

The agency subsequently canceled the meeting. ODAC held a similar meeting on April 27-28 that scrutinized six indications for a handful of checkpoint inhibitors that target programmed death-1 (PD-1) and programmed death-ligand 1 (PD-L1) inhibitors. Three of those indications are being withdrawn from the U.S. market, and one indication has gained full approval.

Company Will Pull Copiktra’s FL Indication

On Dec. 3, Secura Bio said that it would voluntarily withdraw Copiktra’s (duvelisib) U.S. indication for the treatment of adults with relapsed or refractory follicular lymphoma (FL) after at least two systemic therapies. The FDA gave accelerated approval to that indication on Sept. 24, 2018. The capsule also is approved for relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma after at least two therapies.

In its press release announcing the decision, the company said that “after a Secura Bio strategic assessment of Copiktra and subsequent consultation with the U.S. Food and Drug Administration (FDA), the company made the determination that the current treatment landscape for FL patients in the U.S. and the logistics, cost and timing of the post-marketing requirements (PMR) for Copiktra in FL was no longer merited.…This is a business decision and is not related to any changes in either the efficacy or safety associated with Copiktra.”

Secura Bio also said future efforts and resources will focus on “new applications for T-cell lymphoma where initial data appear encouraging.”

The withdrawals come amid scrutiny of the accelerated approval pathway, most notably the FDA’s use of it to approve Biogen and Eisai Co., Ltd.’s Alzheimer’s drug Aduhelm (aducanumab) on June 7. Following the uproar over the decision, including “allegations of an inappropriately close relationship between the FDA and the industry,” FDA Acting Commissioner Janet Woodcock, M.D., asked the HHS Office of Inspector General (OIG) to review the process leading to the approval and whether it was consistent with the FDA’s policies. Instead, OIG said in August that it would undertake a broader review of the accelerated approval pathway, with an issue date expected in 2023.

Pathway Is Useful but Needs Improvement

ODAC and OIG aren’t the only ones analyzing the process. An article published Dec. 2 in Science examined the pathway, maintaining that the Aduhelm decision demonstrates that the FDA “showed a willingness to embrace early approval pathways in ways that risk FDA’s reputation and undermine its core role in keeping the market free of worthless or dangerous medical products.” The authors maintain that “accelerated approval is an important regulatory pathway worth trying to save,” but it needs improvement, particularly with respect to confirmatory trials that support products’ approval.

An article published Aug. 24 in the Journal of Comparative Effectiveness Research outlined 10 possible reforms to the pathway. The authors asserted that condemnations of the FDA’s Aduhelm approval argue that the agency’s use of accelerated approval “has now fully succumbed to a gradual erosion in the standard of evidence deemed adequate for approval. In addition, there are longstanding concerns that the regulatory mechanism to ensure that drug sponsors perform high-quality confirmatory trials is ineffective, undermined by the lack of financial incentives and the apparent lack of will within the FDA to exercise their existing powers to withdraw approval when trials are not conducted expeditiously or fail to confirm the intended clinical benefit.”

And in April, the Institute for Clinical and Economic Review (ICER) published a white paper that outlined potential ways to strengthen the program. According to ICER, “although the majority of accelerated approvals convert to full approval within a reasonable timeframe of 3 years, many products take significantly longer, and those that fail to produce evidence or that have evidence that fails to confirm patient benefit do not always leave the stage quickly. Expedited approval seems to be working in most cases, but the vision of a matching expedited withdrawal has not been realized.”

This story was reprinted from AIS Health’s monthly publication RADAR on Specialty Pharmacy.

Angela Maas

Angela has an extensive background of editing, reporting and writing for trade and consumer publications. She has written Radar on Specialty Pharmacy (formerly called Specialty Pharmacy News) since she joined AIS Health in 2005 and has broad knowledge of the various issues at play within the space. Before joining AIS Health, she was managing editor at Employee Benefit News and Employee Benefit News Canada and managing editor at HemAware (a hemophilia publication), Lupus Living and Momentum (a multiple sclerosis publication). She has a B.A. in English and an M.A. in British literature from Arizona State University.

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