As the Duchenne muscular dystrophy community learns about the early, yet promising, data from Sarepta Therapeutics Inc. on its experimental gene therapy, many affected families may look to the new Right To Try Act as a way to gain early access to the treatment years before it is cleared for the U.S. market, said former biotech executive Michael Becker.
But Sarepta is not providing the gene therapy, known as AAVrh74.MHCK7.micro-Dystrophin, or any of its other experimental treatments to patients under the Right To Try process. Nor is the biotech company allowing use of the medicine under the Food and Drug Administration's expanded access program, also called compassionate use, which allows regulators to help terminally ill patients obtain and use experimental therapies outside of the clinical trial process.
The Right To Try Act, signed into law on May 30 by President Donald Trump, who pushed to get the legislation enacted, permits patients to bypass the FDA's compassionate-use process.
The experimental drugs used under Right To Try must have at least completed the phase 1 testing process, though those studies only check for toxicity, not safety or efficacy.
On June 19, shares of Sarepta soared after the company reported results from its phase 1/2a study of AAVrh74.MHCK7.micro-Dystrophin in three patients with Duchenne muscular dystrophy, or DMD, a rare, debilitating, progressive and ultimately fatal childhood genetic disease caused by mutations in the gene encoding dystrophin, a protein that plays a key role in maintaining muscle integrity.
The early AAVrh74.MHCK7.micro-Dystrophin data demonstrated that the gene therapy produced robust levels of dystrophin. The phase 1/2a data also showed average reduction of more than 87% in the patients' creatine kinase levels on day 60. Creatine kinase is an enzyme associated with muscle damage and patients with Duchenne uniformly exhibit high levels of it.
But Sarepta said it currently was "unable to offer a compassionate-use program on a fair and sustainable basis for any of our investigational agents without jeopardizing our ability to provide patients broad, sustainable and long-term access."
Sarepta said its policy, posted on its website, also applies to anyone seeking access under the Right To Try Act.
"Compassionate use and other mechanisms for pre-approval access to our DMD exon-skipping agents must balance our shared urgency with many other factors," the company said. "These include regulatory and practical considerations, such as each agent's benefit-risk profile, the impact on clinical development, and the feasibility of providing fair and sustainable access."
"In other words, the needs of the many outweigh the needs of the few," said Becker, president and founder of MDB Communications LLC.
Becker applauded Sarepta's transparency in its experimental drugs access statement, "which makes clear why they are focusing solely on the clinical trial route."
"I believe most small [and] mid-size biotech companies share this position and fear for the future as Right To Try advocates realize the false promises of this poorly conceived legislation and how the FDA was never a barrier to early access for experimental treatments," Becker told S&P Global Market Intelligence.
Supporters of the Right To Try Act argued the law was needed because the FDA's compassionate-use process was too slow for some patients, even though the agency has consistently maintained it approves 99% of the 1,000 applications it receives annually, with most given the go-ahead within a few days and emergency requests generally granted immediately over the phone.
The authors of a new analysis published June 15 in the Journal of the American Medical Association said the FDA and the biopharmaceutical industry have established a balance between investigational new drug access and patient safety, which "may be compromised by policymakers seeking to speed access to experimental medicines through the Right to Try Act."
Alison Bateman-House, an assistant professor in the Division of Medical Ethics at New York University Langone Medical Center, also said she sees no advantage for a company to use the Right To Try process.
"A number of companies' access policies emphasize the centrality of FDA review and approval," she told S&P Global Market Intelligence. "This indicates that they will not be using [Right To Try], which does not involve the FDA."
Bateman-House noted that the 21st Century Cures Act, signed by President Barack Obama in December 2016, mandated that biopharmaceutical companies post their compassionate-use policies on their websites within 60 days after the law was enacted.
"The Cures language, which I was involved with, sought to ensure transparency once an investigational product entered phase 2," Bateman-House said.
Before the Cures law was enacted, only 19% of the 100 publicly traded companies examined by Avalere Health in October 2016 posted compassionate-use access policies on their websites.
By March 2017, that number had more than doubled, with 47% of the drugmakers analyzed posting the policies.
The 2016 and 2017 analyses did not examine how many of the companies' policy statements also included language about Right To Try access, given the federal statute had not yet been enacted at that time, though about 40 states had passed their own laws.
Sarepta said it included the Right To Try language in its statement when it originally posted the policy online in February 2017 because of those state laws.
With the Right To Try Act now law, companies may find themselves having to clarify their policies on the matter, given the FDA has advised its staff to direct anyone seeking medicines under the new statute to contact the drug's manufacturer.