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bluebird's (BLUE) BLA for CALD Therapy Gets Priority Review

bluebird's (BLUE) filing for eli-cel, a gene-therapy to treat cerebral adrenoleukodystrophy, gets priority review from the FDA.

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This story originally appeared on Zacks

bluebird bio, Inc. BLUE announced that the FDA has granted priority review to the biologics license application (BLA) seeking approval for elivaldogene autotemcel (eli-cel, Lenti-D) to treat cerebral adrenoleukodystrophy (CALD) in patients aged less than 18 years who do not have a matched donor sibling.

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The regulatory agency has prescribed a target action date of Jun 17, 2022 for the BLA.

We remind investors that earlier this August, bluebird announced that the FDA has placed a clinical hold on the eli-cel clinical program, which is still ongoing. The hold was put because of reported Suspected Unexpected Serious Adverse Reactions of myelodysplastic syndrome likely mediated by Lenti-D lentiviral vector insertion. In the meantime, all patients who received eli-cel in the clinical program continue to be closely monitored, per study protocols.

The BLA so filed is 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 also filed data for 23 subjects dosed in the ongoing phase III ALD-104 study, which has completed enrolment.

Apart from priority review, the therapy has also been granted orphan drug, rare pediatric disease designation and breakthrough therapy designations by the FDA.

The stock has plunged 75.5% so far this year compared with the industry’s 19% decline.

Zacks Investment ResearchImage Source: Zacks Investment Research

CALD is a rare, progressive and irreversible neurodegenerative disorder that affects 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.

Per the company, eli-cel has been developed as a one-time gene-therapy to treat the underlying cause of CALD and stabilize neurologic function. The therapy aims to stop the progression of CALD and consequently preserve as much neurological function as possible.

The therapy has already been approved in the European Union under the trade name Skysona in July 2021. A potential approval by the FDA will make eli-cel the first and only therapy for the treatment of CALD in the United States.

Last month, bluebird announced that the FDA granted priority review to the BLA seeking approval for beti-cel as a potential treatment for β-thalassemia across all ages and genotypes who require regular red blood cell transfusions. A decision from the FDA is expected by May 20, 2022.

An FDA approval to either or both eli-cel and beti-cel will provide a boost to the company’s pipeline, following bluebird’s decision to wind-down European operations to focus on the U.S. market.

Zacks Rank & Other Stocks to Consider

bluebird currently carries a Zacks Rank #2 (Buy). Other top-ranked stocks in the overall healthcare sector include GlaxoSmithKline GSK, IVERIC bio ISEE and Repligen RGEN. While Repligen sports a Zacks Rank #1 (Strong Buy), both GlaxoSmithKline and IVERIC bio presently carry has a Zacks Rank #2. You can see the complete list of today’s Zacks #1 Rank stocks here.

Repligen’s earnings per share estimates for 2021 have increased from $2.76 to $2.90 in the past 60 days. The same for 2022 has increased from $3.03 to $3.21 in the past 60 days. Shares of Repligen have risen 38.5% in the year so far.

Earnings of Repligen beat estimates in all the last four quarters, delivering a surprise of 49.2%, on average.

IVERIC bio’s loss per share estimates for 2021 have narrowed from $1.18 to $1.09 in the past 60 days. The same for 2022 has narrowed from $1.17 to $1.03 in the past 60 days. Shares of IVERIC bio have gained 121% in the year so far.

IVERIC bio’s earnings missed estimates in three of the last four quarters and surpassed expectations once, with the negative surprise being 5.6%, on average.

GlaxoSmithKline’searnings estimates per share for 2021 have increased from $2.82 to $3.05 in the past 60 days. The same for 2022 has increased from $3.08 to $3.25 in the past 30 days. Shares of Glaxo have risen 17.1% in the year so far.

Earnings of GlaxoSmithKlinebeat estimates in three of the last four quarters and missed expectations in one, the average surprise being 15.3%.



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