As lawmakers trudge through debt ceiling negotiations, the possibility that the U.S. will default on its loan obligations in the coming days looms like a dark cloud over Washington. Should government coffers run dry on June 1, as Treasury Secretary Janet Yellen has warned they could, federal workers will go unpaid and agencies like the FDA could see operations come to a screeching halt.

Just last week, FDA Commissioner Robert Califf said that without a bipartisan bill to raise the debt limit by June, the regulator wouldn’t have the money to pay staff and scientific reviewers, potentially delaying upcoming drug approval decisions, advisory committee meetings and regulations.

And with President Joe Biden and House Speaker Kevin McCarthy scrambling to finish negotiations — while haggling over where to cut federal spending in future budget packages — it’s unclear when a deal might be struck, or how long a potential default could last. For patients and pharmaceutical companies depending on the FDA’s actions, a holdup of any length in the decision-making process could be devastating.

And with President Joe Biden and House Speaker Kevin McCarthy scrambling to finish negotiations — while haggling over where to cut federal spending in future budget packages — it’s unclear when a deal might be struck, or how long a potential default could last. For patients and pharmaceutical companies depending on the FDA’s actions, a holdup of any length in the decision-making process could be devastating.

The drug, called Ocaliva, is already marketed as a treatment for primary biliary cholangitis and it could be the first FDA-approved therapy for NASH. However, the FDA’s advisers noted serious concerns with the drug. The panel voted 12 -2, with two abstentions, that the benefits of Intercept’s candidate didn’t outweigh potential risks, including concerns that it induces liver injury. Advisers argued that more information on the long-term impacts to patient health were needed and cautioned the FDA to wait for results of an ongoing phase 3 trial, which isn’t expected to be completed until 2025.

June 22: Sarepta’s Duchene gene therapy
The FDA last week deferred its much-anticipated decision for Sarepta Therapeutic’s Duchenne muscular dystrophy candidate until mid-June, stating that it needed additional time to complete the review. However, if the debt default disrupts the agency’s review process, which may include negotiations around the drug’s label and post-marketing requirements for Sarepta, that decision could be even further delayed.

An FDA ad comm panel voted 8-6 backing the accelerated approval of the gene therapy in early May.

And Sarepta said last week that the agency is considering initially approving the drug, SRP-90001, in patients between ages 4 and 5. Year-end results of an ongoing phase 3 trial could support a broader label in the future, the drugmaker added.

If given the go-ahead, the one-time treatment would provide the first potentially curative option to children with the condition, which causes muscles to slowly waste away.

Reference：
https://www.pharmavoice.com/news/debt-ceiling-default-FDA-pharma/651405/