Bluebird Bio Inc. asked US regulators to approve its gene therapy for sickle cell disease, a potential treatment for the incurable illness that could decide the struggling biotechnology company’s future.
(Bloomberg) — Bluebird Bio Inc. asked US regulators to approve its gene therapy for sickle cell disease, a potential treatment for the incurable illness that could decide the struggling biotechnology company’s future.
Bluebird’s application for Food and Drug Administration clearance of the therapy, lovo-cel, follows a series of safety scares and delays that pushed the filing by more than a year. Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG already submitted an application for a rival gene-editing drug earlier this month.
The shares rose 6.7% at 7:32 a.m. in New York on Monday. The stock had declined about 50% this year through the close on April 21.
An inherited condition caused by a gene mutation, sickle cell disease affects about 100,000 Americans, with Black people among those at highest risk. The faulty gene causes blood cells to become misshapen into a characteristic crescent or sickle shape that can slow or halt blood flow, leaving patients in extreme pain.
Bluebird’s lovo-cel introduces a correct copy of the flawed gene into patients’ cells. The treatment resolved pain crises for all 25 subjects evaluated in a clinical trial, yet still faces questions over potential risks.
A few people who took the drug in studies developed illnesses that looked like cancer or the beginnings of the disease, creating concern about the treatment’s safety and dealing Bluebird a huge setback. The company later determined only one of the cases was actually cancerous and was unrelated to the drug itself. The fallout could still weigh on lovo-cel if it’s approved, especially since the rival treatment from Vertex and Crispr Therapeutics hasn’t faced similar questions.
Bluebird needs lovo-cel to succeed after nearly running out of money last year. Two other rare-disease therapies approved last year — Zynteglo for a blood disorder and Skysona for a brain disease — helped rebuild confidence in the company, but they’re unlikely to bring in enough to help Bluebird’s cash woes.
Lovo-cel will also have to contend with more conventional treatments, like Pfizer Inc.’s newly acquired Oxbryta. Pfizer gained the treatment, which reduces sickling and eases blood flow, in its $5.4 billion purchase of Global Blood Therapeutics Inc. last year.
While potentially curative, the genetic therapies from Bluebird and Vertex will likely be limited to the sickest patients because of their intensity. People must spend weeks in the hospital receiving harsh chemotherapy to prepare them to receive their modified cells.
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