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FDA rejects Abeona cell therapy, asks for more manufacturing data

The Food and Drug Administration has rejected an experimental cell therapy for a rare skin condition, asking its developer, Abeona Therapeutics, to provide more information about how the treatment is made.

Abeona on Monday said the FDA issued a so-called complete response letter for the therapy, known as prademagene zamikeracel, or pz-cel. The company had sought approval for its use treating a form of epidermolysis bullosa, or EB.

Testing showed Abeona’s therapy — a one-time treatment made from a person’s skin cells — can help heal wounds, and lower pain in people with a severe recessive dystrophic form of the disease. According to Abeona, however, the FDA turned back the company’s application over questions related to pz-cel’s manufacturing. The agency hasn’t asked the company to run another trial, Abeona said.

To satisfy the FDA’s concerns, Abeona has to meet FDA requirements for manufacturing as well as so-called release testing, which ensures the purity and safety of a pharmaceutical product. In recent meetings with the regulator, Abeona said it had intended to provide manufacturing data prior to approval and full “validation” reports about its production processes by the middle of the year. The proposed timing wouldn’t allow the FDA to review those reports by a May 25 decision deadline, though, leading the agency to reject Abeona’s application.

In a statement, Abeona CEO Vishwas Seshadri said the company plans to resubmit an application in the third quarter, after which a new review process would start. On a Tuesday conference call with analysts, Seshadri added the FDA has indicated it won’t take the full six months to evaluate Abeona’s application once it’s resubmitted.

“The FDA has provided very clear instructions on what their expectations are,” he said, and the agency “did assure us that they’re going to expeditiously review” an eventual filing. That the regulator didn’t mandate a new trial is “huge,” Seshadri added.

Nonetheless, the FDA rejection delays the arrival of a new treatment for EB, which makes the skin fragile and susceptible to frequent blistering.

EB is typically managed by supportive care. Recently, genetic medicines have emerged as a therapeutic option. A topical gene therapy from Krystal Biotech was approved last year for people with the dystrophic form of the disease.

Abeona is one of several gene and cell therapy developers to hit manufacturing-related regulatory setbacks in recent years. The February clearance of a first-of-its-kind cancer cell therapy from Iovance Biotherapeutics came after multiple holdups, for instance. Krystal’s gene therapy, as well as other approved treatments from Bluebird Bio and Sarepta Therapeutics, also faced longer development timelines due to questions about production processes.