RCKT Stock Up as FDA Accepts Resubmitted BLA for Gene Therapy

Rocket Pharmaceuticals RCKT announced that the FDA has accepted the resubmitted biologics license application (BLA) seeking approval for its gene therapy Kresladi (marnetegragene autotemcel; marne-cel) to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder.

A decision from the FDA on the BLA is expected on March 28, 2026.

Shares of Rocket Pharmaceuticals were up 23.1% yesterday following the announcement.

The BLA was based on positive data from a global phase I/II study, which showed that treatment with Kresladi led to 100% overall survival for all LAD-I patients at 12 months post-infusion, thereby meeting all primary and secondary endpoints of the study.

Treatment with Kresladi was well tolerated, with no treatment-related serious adverse effects reported.

If Kresladi is eventually approved by the FDA, the company will be eligible for a Rare Pediatric Disease Priority Review Voucher (PRV) — an incentive given by the FDA to encourage the development of drugs and biologics for rare and serious diseases.

If any company gets FDA approval for a new drug for a rare genetic disorder, it can receive a PRV. It can then use that voucher to expedite the approval of another drug or sell it to a large pharmaceutical company for a cash payment.

RCKT's Price Performance

Year to date, shares of Rocket Pharmaceuticals have plunged 66.1% against the industry’s increase of 7.7%.

Earlier CRL for RCKT's Kresladi BLA

Last June, the FDA issued a complete response letter (CRL) to the BLA for Kresladi for treating LAD-I.

Per the CRL, the FDA sought "limited" additional information on the Chemistry Manufacturing and Controls (CMC), which was part of the BLA submission.

Notably, this was the second time that the FDA had requested additional information from the company on Kresladi’s CMC. In February 2024, the FDA extended the review period of the Kresladi BLA by an additional three months.

LAD-I is a rare, severe pediatric genetic disorder that is caused by a defect in the ITGB2 gene. This leads to a deficiency in CD18 and can cause recurrent and life-threatening infections, fatally affecting the immune system.

The only curative treatment for LAD-I is a bone marrow transplant, which is often not readily available and carries substantial morbidity and mortality risk.

With the FDA accepting the resubmitted BLA for Kresladi, a potential approval of the same would mark RCKT’s first commercial product and provide a steady source of revenues.

Rocket Pharmaceuticals, Inc. Price

Rocket Pharmaceuticals, Inc. price | Rocket Pharmaceuticals, Inc. Quote

RCKT's Zacks Rank & Stocks to Consider

Rocket Pharmaceuticals currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the biotech sector are Chemomab Therapeutics CMMB, ANI Pharmaceuticals ANIP and Alnylam Pharmaceuticals ALNY, each carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

In the past 60 days, estimates for Chemomab Therapeutics’ 2025 loss per share have narrowed from $2.40 to 60 cents. Loss per share estimates for 2026 have narrowed from $2.80 to $1.00 during the same period. CMMB stock has plunged 52.4% year to date.

Chemomab Therapeutics’ earnings beat estimates in three of the trailing four quarters, while meeting the same on the remaining occasion, with an average surprise of 26.25%.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2025 earnings per share have risen from $7.25 to $7.29. During the same period, earnings per share for 2026 have increased from $7.74 to $7.79. Year to date, ANIP’s shares have rallied 60.5%.

ANI Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with an average surprise of 22.66%.

In the past 60 days, estimates for Alnylam’s 2025 earnings per share have risen from $3.35 to $3.98. During the same period, earnings per share for 2026 have increased from $8.95 to $9.61. Year to date, ALNY’s shares have surged 99.5%.

Alnylam’s earnings beat estimates in three of the trailing four quarters, while meeting the same on the remaining occasion, with an average surprise of 348.36%.

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This article originally published on Zacks Investment Research (zacks.com).

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