Bristol Myers wins, Apellis loses in busy week at Europe's drug regulator

Shortly after Europe’s Committee for Medicinal Products for Human Use (CHMP) recommended refusal of a marketing authorization for Apellis’ geographic atrophy (GA) drug Syfovre, the company said it will appeal the guidance.

On the positive side of the ledger, the European Medicines Agency committee recommended marketing authorizations for four products, including a label expansion for Bristol Myers Squibb's multiple myeloma CAR-T Abecma.

The thumbs down for Apellis' groundbreaking drug—which became the first treatment for GA upon its approval by the FDA in February of last year—was not a surprise. Last month, Apellis revealed that a panel of experts voted down the treatment after an “oral explanation meeting,” triggering a 17% slide in the company’s share price.

Apellis' drug stumbled soon after its U.S. launch because of reports of vascular retinitis. The CHMP determined that the benefits of using Syfovre don't outweigh the risks.

“Although the studies showed that Syfovre slowed the growth of geographic atrophy lesions, this did not lead to clinically meaningful benefits for patients,” the CHMP concluded. “It was noted that benefits of a treatment should impact patients’ everyday functioning, and this was not demonstrated in the studies.”

For its part, the Massachusetts-based company pointed to results from two phase 3 trials which showed that the treatment reduced GA lesion growth, with treatment benefits accumulating over time. 

“We are very disappointed in this outcome on behalf of the millions of Europeans with GA who currently have no treatment options for this irreversible form of blindness,” Jeffrey Eisele, Ph.D., Apellis’ chief development officer, said in a statement. “We plan to promptly initiate the re-examination of our application and address CHMP questions.”

BMS scores with Abecma

Meanwhile, the CHMP’s positive opinion on BMS’s Abecma is the first in earlier lines of treatment for a CAR-T cell therapy for multiple myeloma, according to the drugmaker. 

CHMP specifically recommended the drug for patients who've tried two prior therapies, as opposed to the drug's initial EU nod in the fourth line of treatment.

Samit Hirawat, M.D., BMS’s chief medical officer, called the endorsement an “important milestone.”

“(It) moves us closer to offering a first-in-class, personalized therapy to patients in Europe battling this incurable disease after exhausting prior treatment options with the three standards of care.”

Also gaining recommendations for label extensions were Apellis and Sobi's PNH drug Aspaveli, Pfizer's pneumococcal vaccine Prevnar 20 and Eli Lilly's thyroid cancer drug Retsevmo. 

CHMP gives thumbs up to 3 products

The CHMP recommended a marketing authorization for Allecra’s Exblifep (cefepime/enmetazobactam) for urinary tract infections accompanied by hospital-acquired pneumonia and for the treatment of patients with bacteria in the bloodstream. The antibiotic combination treatment also is under review by the FDA.

Also getting a positive recommendation from the committee was Evive Biotech and Acrotech Biopharma’s Ryzneuta (efbemalenograstim alfa) for neutropenia and febrile neutropenia caused by chemotherapy. The FDA signed off on the leukocyte growth factor two months ago.

The CHMP also said yes to Neuraxpharm’s schizophrenia medicine Niapelf (paliperidone), which is the generic version of Johnson & Johnson’s Invega Sustenna, which was approved in the U.S. in 2009 and in Europe as Xeplion in 2011.

CHMP recommends rejection of Nezglyal

Minoryx Therapeutics’ Nezglyal (leriglitazone) got a negative recommendation from the committee. The drug is for the treatment of the genetic condition cerebral adrenoleukodystrophy, which damages the brain and spinal cord. The Spanish company gained FDA fast-track designation for the treatment in 2020.