I'm launching an investigation into why the FDA is denying access to a drug that has been helping patients with Duchenne muscular dystrophy for years.
Investigate why the FDA is denying access to ataluren for patients with Duchenne muscular dystrophy.
Occurrences
Evidence
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The article says Ron Johnson said in March that he was investigating the FDA for rejections of rare disease treatments.
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Axios says Sen. Ron Johnson has been probing the FDA's rejection of rare disease drugs and that his concern dates back years.
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Within the June 9-July 8 lookback window, the report says FDA agreed to reverse a rejection of Regenxbio's rare-disease gene therapy and that the action was part of a broader series of rare-disease review reversals under new leadership. It does not identify ataluren, Translarna, Duchenne access, or a completed Johnson ataluren investigation.
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Axios reports that Sen. Ron Johnson, chair of the Senate Permanent Subcommittee on Investigations, said he was probing FDA rejections of rare-disease drugs and had concerns dating back to 2017. The story frames this as ongoing scrutiny of FDA rare-disease decision-making, not as a completed ataluren-specific investigation or delivered patient access.
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Congress.gov lists Sen. Ron Johnson as sponsor of S.204 and records that it became Public Law No. 115-176 on May 30, 2018. CRS summarizes the law as exempting provision of certain unapproved investigational drugs to terminally ill patients who exhausted approved options and cannot join a clinical trial.
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EMA states that its human medicines committee confirmed a recommendation not to renew Translarna (ataluren) authorization for Duchenne muscular dystrophy because effectiveness had not been confirmed. EMA also notes Translarna had been authorized in the EU for DMD caused by nonsense mutation and that the review considered post-authorization studies, registries, patients, caregivers, clinicians, and organizations.
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UNII availability does not imply any regulatory review or approval.
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Further development of Translarna for the treatment of nmDMD in the United States is not planned.
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we will continue to provide ataluren for individuals currently on treatment while existing supply lasts.
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I’m launching an investigation into why the FDA is denying access
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introduced the Right to Try for Individualized Treatments Act
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Date Approved May 30, 2018
Assessments
Johnson materially advanced related rare-disease and investigational-drug access policy by sponsoring the federal Right to Try Act, which became law in 2018, and later reporting shows he has continued probing FDA rare-disease drug rejections. However, the evidence does not show that he completed an ataluren-specific FDA investigation, produced findings explaining denial of access, or secured ataluren access for Duchenne muscular dystrophy patients. Because the record supports serious related oversight and legislative effort but not full delivery of the specific ataluren promise, partial credit is appropriate.
The available evidence shows Johnson was actively probing the FDA’s rejection of rare-disease treatments during the same Senate term, which is meaningful oversight effort consistent with the promise. But the record here does not clearly show a completed ataluren-specific investigation, formal findings, or a resolved access outcome for Duchenne muscular dystrophy patients. That supports partial credit rather than full delivery.