5 Best Mid-Cap Growth Stocks To Buy Now

2. Sarepta Therapeutics Inc (NASDAQ:SRPT)

Number of Hedge Fund Investors: 50

Earlier this month, Citi started covering Sarepta Therapeutics Inc (NASDAQ:SRPT) with a Buy rating. Citi likes Sarepta Therapeutics Inc’s (NASDAQ:SRPT) risk/reward profile heading into FDA’s review of Sarepta Therapeutics Inc’s (NASDAQ:SRPT) drug Elevidys for full approval for the treatment of Duchenne muscular dystrophy.

A total of 50 hedge funds tracked by Insider Monkey had stakes in Sarepta Therapeutics Inc (NASDAQ:SRPT). The most significant stakeholder of Sarepta Therapeutics Inc (NASDAQ:SRPT) was Kurt Von Emster’s VenBio Select Advisor which owns a $488 million stake in Sarepta Therapeutics Inc (NASDAQ:SRPT).

Bronte Capital Amalthea Fund made the following comment about Sarepta Therapeutics, Inc. (NASDAQ:SRPT) in its Q3 2023 investor letter:

“The FDA is widely considered to be the world’s foremost regulator of drug products, with a stringent and rigorous process for evaluating new marketing applications. Disagreements between the FDA and regulators in other developed markets (such as the European Medicines Agency or the Australian Therapeutic Goods Administration (TGA)) are rare, and when they do occur, it is usually because the FDA has taken a more critical view of the applicant’s evidence.

For a drug to be approved in the US, it must meet the statutory requirement of “substantial evidence of effectiveness” under the Federal Food, Drug, and Cosmetic Act. There are essentially three ways to meet this requirement. Normally, the FDA expects the sponsor to succeed in two “adequate and well-controlled studies”. Alternatively, the sponsor can rely on success from a single study if the results from that study are “very persuasive”, or if they are combined with some sort of independent confirmatory evidence. For the most part lobbying from the cohort of patients, the “patient voice”, has played a relatively minor role in the FDA’s decision-making process and the agency has been prepared to make tough but rational decisions when the “substantial evidence” standard is clearly not met.

However, this was not the case in 2016 when the FDA famously overruled its own review team and external advisory committee to approve Sarepta Therapeutics, Inc.’s (NASDAQ:SRPT) controversial drug for Duchenne muscular dystrophy (Exondys 51). At the time, Sarepta had completed a single phase 2 trial in just 12 patients which, per the FDA Commissioner (Robert Califf) himself, had “major flaws” in both its design and conduct. Ellis Unger, director of the Office of Drug Evaluation at the FDA, declared that the drug was a “scientifically elegant placebo”, and that patients and their families were taking on unknown risks for likely non-existent benefits…” (Click here to read the full text)