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FDA Committee Recommends bluebird's (BLUE) CALD Therapy

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bluebird bio BLUE announced that the  FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) has unanimously recommended approval of its gene therapy candidate elivaldogene autotemcel (eli-cel) to treat cerebral adrenoleukodystrophy (CALD), a rare neurodegenerative disease.

The CTGTAC voted 15-0, unanimously recommending the benefits of treatment with eli-cel to outweigh the risks. The FDA Committee also voted 13-1, concluding the safety data from lovo-cel, another gene therapy developed for treating sickle-cell disease, is not relevant to review for eli-cel.

If approved by the regulatory agency, eli-cel will be the first approved gene therapy to treat early active CALD, in the United States.

bluebird bio’s biologics license application (BLA) filed with the FDA seeks approval for eli-cel to treat early CALD in patients less than 18 years of age, who do not have a matched sibling hematopoietic stem cell (HSC) donor. The filing was granted priority review by the FDA last December and the regulatory agency’s final decision on the gene therapy is expected by Sep 16, 2022.

Shares of bluebird bio have plunged 62.7% in the year so far compared with the industry’s 25.4% decline.

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The CTGTAC’s positive recommendation was based on data from the completed phase II/III Starbeam study (ALD-102) wherein 90.6% of the patients achieved the primary endpoint of Major Functional Disabilities-free survival at 24 months. bluebird bio also filed data for subjects dosed in the ongoing phase III ALD-104 study, which completed enrolmentand treatment of all patients.

CALD is a rare, progressive and irreversible neurodegenerative disorder affecting young children. It involves the breakdown of myelin, the protective sheath that nerve cells need for neurologic function. Patients who do not receive treatment for CALD die within five years of symptom onset. Currently, allogeneic hematopoietic stem cell transplant (allo-HSCT) is the only effective treatment option but it carries a significant risk for patients.

Eli-cel is developed to treat the underlying cause of CALD and stabilize the neurologic function. The therapy aims to stop the progression of CALD and preserve as much neurological function as possible.

The CTGTAC is also set to give its recommendation on another bluebird bio regulatory filing, seeking approval for betibeglogene autotemcel (beti-cel) as a potential treatment for β-thalassemia. The meeting will be scheduled on Jun 10.

Presently, BLUE has no approved/marketed drug in its portfolio. A tentative approval for either beti-cel or eli-cel will provide bluebird bio with its first FDA-approved drug.

bluebird bio is currently facing the risk of a severe cash crunch. In fact, management expressed concerns over BLUE’s capacity to continue as a going concern due to the pressure on its existing cash balance, which is expected to dry up in the near future.

An approval of either or both beti-cel and eli-cel will also make bluebird bio eligible for priority review vouchers (PRVs) from the FDA. BLUE anticipates selling these vouchers to boost its cash resources.

In a separate press release, bluebird bio also announced that the NASDAQ halted trading BLUE’s common shares on Jun 9 and 10. This is likely to take into account for the impact of the CTGTAC’s recommendations on BLUE’s share price. Shares are expected to rise once trading resumes on Jun 11 after the FDA committee’s positive recommendation.

bluebird bio, Inc. Price

bluebird bio, Inc. Price
bluebird bio, Inc. Price

bluebird bio, Inc. price | bluebird bio, Inc. Quote

Zacks Rank & Stocks to Consider

bluebird bio carries a Zacks Rank #3 (Hold) at present. Some better-ranked stocks in the overall healthcare sector are Abeona Therapeutics ABEO, Alkermes ALKS and Sesen Bio SESN. While Sesen Bio sports a Zacks Rank #1 (Strong Buy) at present, both Abeona Therapeutics and Alkermes carry a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Sesen Bio’s loss per share estimates for 2022 have declined from 33 cents to 32 cents in the past 60 days. Shares of SESN have inched up 1.7% in the year-to-date period.

Earnings of Sesen Bio beat estimates in three of the last four quarters and missed the mark on one occasion, the average surprise being 69.9%. In the last reported quarter, SESN delivered an earnings surprise of 100%.

Alkermes’ loss per share estimates for 2022 have narrowed from 14 cents to 3 cents in the past 60 days. Shares of ALKS have risen 20.9% year to date.

Earnings of Alkermes beat estimates in each of the last four quarters, the average being 350.5%. In the last reported quarter, ALKS delivered an earnings surprise of 1,100%.

Abeona Therapeutics’ loss per share estimates for 2022 have narrowed from 34 cents to 31 cents in the past 30 days. The same for 2023 has narrowed from 15 cents to 13 cents in the same time frame. Shares of ABEO have plunged 49.6% in the year-to-date period.

Earnings of Abeona Therapeutics missed estimates in two of the trailing four quarters and met the same on the remaining two occasions, the average negative surprise being 8.2%. In the last reported quarter, ABEO missed on earnings by 25%.


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