FDA seeks additional data for Rocket Pharma’s gene therapy By Investing.com

CRANBURY, N.J. – Rocket Pharmaceuticals, Inc. (NASDAQ: NASDAQ:) has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) regarding its Biologics License Application for KRESLADI™, a gene therapy designed to treat severe leukocyte adhesion deficiency-I (LAD-I). The FDA has requested additional Chemistry Manufacturing and Controls (CMC) information to complete its review.

Following discussions with FDA senior leaders from the Center for Biologics Evaluation and Research (CBER), Rocket Pharmaceuticals is working to provide the additional CMC information to support the approval of KRESLADI™. “It is reassuring to have the FDA as a close collaborator,” stated Gaurav Shah, M.D., CEO of Rocket Pharma, expressing optimism for the therapy’s timely advancement due to the FDA’s cooperative stance.

KRESLADI™, a lentiviral vector-based gene therapy, has shown promising results in a global Phase 1/2 study, with all nine LAD-I patients surviving 12 months post-infusion and no treatment-related serious adverse events reported.

The therapy has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric, and Fast Track designations, as well as PRIME and Advanced Therapy Medicinal Product (ATMP) designations in the EU.

Severe LAD-I is a rare genetic disorder that compromises the immune system’s ability to fight infections. KRESLADI™ aims to provide a functional copy of the ITGB2 gene to address the root cause of the disease. Currently, the only potential curative treatment is a bone marrow transplant, which has significant risks and may not be available for all patients.

Rocket Pharmaceuticals is a late-stage biotechnology company focused on developing genetic therapies for rare disorders. Its pipeline includes programs for Fanconi Anemia, Pyruvate Kinase Deficiency, and cardiovascular diseases such as Danon Disease. This news is based on a press release statement from Rocket Pharmaceuticals.

In other recent news, Rocket Pharmaceuticals has seen several key developments. The biotechnology firm has successfully amended its charter to increase the number of authorized common shares from 120 million to 180 million.

This change, which was approved by shareholders at the Annual Meeting, could provide Rocket Pharmaceuticals with the flexibility for future corporate needs, including financing activities and strategic initiatives.

In addition, Rocket Pharmaceuticals has been granted orphan medicinal product designation by the European Commission for its gene therapy candidate RP-A601. This therapy is designed to treat plakophilin-2 related arrhythmogenic cardiomyopathy, a rare and life-threatening heart disease.

Furthermore, the company has reported sustained efficacy in gene therapy trials, including treatments for severe Leukocyte Adhesion Deficiency-I, Fanconi Anemia, and Pyruvate Kinase Deficiency.

Coverage on Rocket Pharmaceuticals was recently initiated by Goldman Sachs with a neutral rating. The firm highlighted the potential of the company’s leading asset, RP-A501. Rocket Pharmaceuticals has also announced the appointment of Aaron Ondrey as Chief Financial Officer, along with other strategic internal promotions, to strengthen its transition to commercial operations and advance its treatment pipeline.

These recent developments underscore the company’s ongoing efforts in the field of gene therapy and its commitment to addressing rare and life-threatening diseases.

InvestingPro Insights

As Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) navigates the regulatory pathway for KRESLADI™, its financial health and market performance provide additional context for investors monitoring the situation. Despite the recent setback with the FDA’s request for more information, the company’s balance sheet reflects a strategic position where it holds more cash than debt, an indicator of financial stability that could be crucial as it addresses the FDA’s requests.

InvestingPro data shows a market capitalization of $1.95 billion and a Price/Book ratio for the last twelve months as of Q1 2024 at 4.41. This ratio suggests that the market values the company higher than its net asset value, which could be attributed to investor expectations for future growth or the company’s intellectual property value, including its pipeline of gene therapies.

Moreover, five analysts have revised their earnings upwards for the upcoming period, as per InvestingPro Tips, signaling a potential positive sentiment around the company’s future financial performance. This optimism could be reflective of the market’s confidence in Rocket Pharmaceuticals’ ability to overcome regulatory hurdles and bring its therapies to market.

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