Scott Gottlieb Expects the FDA to Approve 40 Gene Therapies by 2022

(Photo: Eduardo Munoz/Reuters)

FDA commissioner, Scott Gottlieb said that he expects the FDA to approve 40 gene therapies by 2022 in a fireside chat at the 2018 BIO International Convention. 

Gottlieb was interviewed by BIO president and CEO, Jim Greenwood as the event was wrapping up yesterday, and told him that we might see cures for diseases like sickle cell anaemia within 10 years. 

In their wide range discussion, Gottlieb mentioned that the agency will need to approach gene therapy approvals differently in the future, as there are complexities in advanced therapies that are related to product issues as compared to clinical issues. 


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He also indicated that the FDA will publish six guidance documents regarding gene therapies in the near future. 

Another point they discussed, was patient-focused reimbursement. One example is Spark Therapeutics’ refund program, which is an effort to justify its high price for Luxturna — which has an eye-popping price tag of $850,000 — where the company will refund the money if the gene therapy doesn’t work for the patient. 

The FDA commissioner told Greenwood that the agency is working on a formal structure to solicit patient input, which will allow them to develop a guidance framework.