FDA Approves Therapy For Rare Disease Without Randomized Trial Data

Authored by Zachary Stieber via The Epoch Times (emphasis ours),

The Food and Drug Administration on Aug. 14 approved a therapy for a rare disease called recurrent respiratory papillomatosis (RRP).

The U.S. Food and Drug Administration (FDA) in White Oak, Md., on June 5, 2023. Madalina Vasiliu/The Epoch Times

Regulators cleared Papzimeos, an immunotherapy made by Precigen, citing data from a single-arm, open-label trial that looked at how it performed in adults with RRP and who needed at least three surgeries a year.

Patients received four injections of the therapy over 12 weeks following surgical procedures.

About half of the participants needed no surgery in the year following treatment. The safety profile was also deemed favorable.

“Randomized trials are not always needed to approve medical products and this approval is proof of that philosophy,” Dr. Vinay Prasad, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.

“The FDA will always demand the correct clinical study for the specific medical product and disease. Our requirements for products given to tens of millions of healthy people will be different than products given to at most hundreds or thousands of patients with unique diseases.”

The approval is the first announcement from Prasad after he rejoined the FDA following his July resignation.

Prasad had received criticism for not approving enough new drugs and therapies, including from the editorial board of the Wall Street Journal, which said that Prasad “has long criticized such single-arm studies that have no placebo groups.”

Replimune’s treatment for advanced melanoma is among the drugs the FDA has rejected since Prasad became its top vaccine and biologics official.

Prasad said in May that he favors randomized clinical trials but that for some rare diseases, it would be difficult or even impossible to complete such trials. He said that the FDA would have a “flexible regulatory standard” that takes into account “the context of a disease.”

In June, in an article co-authored by FDA Commissioner Dr. Marty Makary, he said that for some products targeting rare diseases, “premarket randomized trials may not be feasible.”

RRP, which is caused by human papillomavirus, features the development of noncancerous tumors in the air passages inside the body. Without removal, the tumors lead to problems such as difficulty breathing.

People with the disease typically undergo multiple surgeries per year because the tumors often regrow after removal.

About 1,000 new cases of RRP are diagnosed in the United States on an annual basis, according to the FDA.

Before the approval of Papzimeos, there were no approved therapies for RRP.

“For more than a century, since RRP was first recognized as a distinct disease, patients have had to rely on repeated surgeries to manage this relentless condition,” Precigen CEO Helen Sabzevari said in a statement. “Today marks a historic turning point.”