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FDA Accepts Vertex (VRTX), CRISPR's BLA Filings for Exa-Cel

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Vertex Pharmaceuticals (VRTX - Free Report) and CRISPR Therapeutics (CRSP - Free Report) announced that the FDA has accepted their biologics license applications (“BLAs”) for exagamglogene autotemcel (“exa-cel”) to treat sickle cell disease (“SCD”) and transfusion-dependent beta-thalassemia (“TDT”).

The FDA granted priority review to the BLA filing for exa-cel in SCD indication, while the exa-cel filing in TDT indication has been accepted for a standard review by the agency. A final decision on the BLAs for exa-cel in SCD and TDT indications are expected by Dec 8, 2023 and Mar 30, 2024, respectively.

Unlike standard reviews that require a time of 12 months for completion, priority reviews shorten the FDA's review timeline to eight months.

The BLA submission is supported by data from the ongoing phase III studies — CLIMB-111 and CLIMB-121 — and an ongoing long-term follow-up study, CLIMB-131.

Exa-cel is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy, developed to treat SCD or TDT. The acceptance of the above BLA filings marks the first time that the FDA accepted gene-editing therapies developed using CRISPR technology for review.

Vertex and CRISPR Therapeutics also filed similar submissions on exa-cel in Europe, which were validated earlier this year in January.

In the year so far, shares of Vertex have increased 15.2% while the same for CRISPR Therapeutics have surged 54.0%. During the same period, the industry has declined 7.6%.

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Vertex has a portfolio of marketed products all of which are approved in cystic fibrosis (“CF”) indication. These are Trikafta/Kaftrio (elexacaftor/tezacaftor/ivacaftor and ivacaftor), Symdeko/Symkevi (tezacaftor/ivacaftor and ivacaftor), Orkambi (lumacaftor/ivacaftor) and Kalydeco (ivacaftor). Apart from exa-cel, it is also developing treatments for neuropathic pain, APOL1-mediated kidney disease, type I diabetes and alpha-1 antitrypsin deficiency.

CRISPR Therapeutics does not have any marketed drugs in its portfolio. Apart from exa-cel, CRSP is currently developing two gene-edited allogeneic CAR-T cell therapy candidates, CTX110 and CTX130, targeting hematological and solid tumor cancers.

If exa-cel BLA is approved, it will be the first marketed product in CRISPR Therapeutics’ portfolio. The FDA nod to exa-cel will also make it Vertex’s first marketed product approved in a non-CF indication. Vertex expects exa-cel to be its next commercial launch.

Apart from exa-cel, VRTX and CRSP are developing stem cell therapies for diabetes. The companies have completed dosing study participants in the phase I study evaluating the safety, tolerability and immune evasion of VCTX210 in type I diabetes (T1D). The company is currently enrolling study participants in a phase I/II study evaluating VCTX211, another stem-derived candidate for T1D indication. This March, Vertex signed another agreement with CRISPR Therapeutics to accelerate the development of the former’s hypoimmune cell therapies for T1D.


Zacks Rank & Stocks to Consider

Vertex and CRISPR Therapeutics currently carry a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include Novo Nordisk (NVO - Free Report) and Novartis (NVS - Free Report) . While Novo Nordisk sports a Zacks Rank #1 (Strong Buy), Novartis carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, the consensus estimate for Novo Nordisk’s 2023 and 2024 earnings per share has increased from $4.51 to $5.07 and $5.26 to $5.91, respectively. Shares of Novo Nordisk are up 17.8% in the year-to-date period.

Earnings of Novo Nordisk beat estimates in two of the last four quarters, met the mark one occasion while missing the mark on another. On an average, the company witnessed an average earnings surprise of 0.35%. In the last reported quarter, Novo Nordisk’s earnings met estimates.

In the past 60 days, estimates for Novartis’ 2023 and 2024 earnings per share have increased from $6.57 to $6.72 and $7.08 to $7.26, respectively. Shares of Novartis are up 11.0% in the year-to-date period.

Earnings of Novartis beat estimates in each of the last four quarters, witnessing an average earnings surprise of 5.15%. In the last reported quarter, Novartis’ earnings beat estimates by 10.32%.

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