Sarepta gene therapy vector gets FDA platform designation

CAMBRIDGE, Mass. - Sarepta Therapeutics, Inc. (NASDAQ:SRPT) announced that its rAAVrh74 viral vector, used in the investigational gene therapy SRP-9003 for limb-girdle muscular dystrophy type 2E/R4, has received platform technology designation from the U.S. Food & Drug Administration (FDA). This designation is a nod to the technology’s potential for reproducibility and adaptability in multiple therapeutic programs.

Louise Rodino-Klapac, Ph.D., the company’s chief scientific officer, highlighted that this is one of the first programs to receive such recognition from the FDA. She emphasized the consistency of data across clinical programs using this vector and the company’s commitment to developing treatments for patients with rare genetic diseases. The company’s strong liquidity position, with a current ratio of 4.02, supports its ambitious research and development initiatives.

The FDA’s platform technology designation aims to expedite drug development, manufacturing, and review processes by allowing sponsors to use prior data from the platform to support new drug applications. To qualify, a technology must be well-understood, reproducible, and capable of supporting the development of multiple drugs or biologics without compromising quality, safety, or manufacturing processes.

SRP-9003 (bidridistrogene xeboparvovec) is designed to deliver a full-length beta-sarcoglycan transgene to skeletal, diaphragm, and cardiac muscle. It is seen as a promising treatment for neuromuscular diseases, particularly for patients with limb-girdle muscular dystrophy Type 2E, many of whom face life-threatening pulmonary or cardiac complications.

Sarepta Therapeutics is focused on genetic medicine for rare diseases, holding leadership positions in therapies for Duchenne muscular dystrophy and limb-girdle muscular dystrophies. The company’s portfolio spans muscle, central nervous system, and cardiac diseases.

This announcement is based on a press release statement and comes amid the company’s efforts to leverage its platform technology for the advancement of rare disease therapies. With impressive revenue growth of 59% over the last twelve months and analysts maintaining a bullish consensus, the company shows promise despite recent stock volatility. However, it is important to note that the success in clinical trials does not guarantee later success, and all potential risks are outlined in the company’s filings with the Securities and Exchange Commission. For deeper insights into Sarepta’s financial health and growth prospects, including exclusive ProTips and comprehensive analysis, visit InvestingPro.

In other recent news, Sarepta Therapeutics has released promising data from its ENDEAVOR study, demonstrating significant protein expression in young patients treated with ELEVIDYS for Duchenne muscular dystrophy. The study showed a mean protein expression of 93.87% of normal, with a consistent safety profile, building on earlier successful results. Additionally, Sarepta announced the Japanese Ministry of Health’s approval of ELEVIDYS for children aged 3 to less than 8, marking the first global approval for patients under 4 years old. This approval is part of Sarepta’s collaboration with Roche, which includes significant milestone payments and royalties outside the U.S.

Meanwhile, Morgan Stanley reiterated its Overweight rating with a $113 price target for Sarepta, following the Japanese approval, suggesting it could alleviate investor concerns about the drug’s safety and market potential. In contrast, Evercore ISI downgraded Sarepta’s stock from "Outperform" to "In Line," reducing the price target to $50, citing competitive pressures and regulatory uncertainties. Evercore ISI also noted potential acceleration in U.S. sales for ELEVIDYS by the second half of 2025, though they anticipate a subsequent decline.

Goldman Sachs maintained its Buy rating with a $100 target, emphasizing Sarepta’s commercial execution and potential risks to ELEVIDYS’ approval for non-ambulatory patients. Sarepta remains focused on diversifying its pipeline, projecting $13 billion in free cash flow by the end of 2030, with anticipated contributions from new programs.

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