It is Memorial Day Weekend! The opening bell for summer vacations has arrived. It is time to do your wardrobe switch – get out the white shoes, sandals and linen clothing and put the corduroys away until Labor Day. Time to wince about last year’s bathing suit and the fact that it appears to have shrunk while stored away. And sorry to say this was a busy week, leaving no time for an intermittent posting between Weekly Roundups, but will shoot for at least one next week. Meanwhile a bit of what happened in this week as everyone is poised to get away or stay home and usher in the summer with a barbeque:
- Gottlieb Signals New Facilitation of Gene Therapy – In a speech given at the annual board meeting of the Alliance for Regenerative Medicine, Dr. Gottlieb stated that we have arrived “at a key point when it comes to cell and gene therapy” – moving from theory to therapy. He pointed up that the agency has put into place a new policy framework in 2017 putting in place an informed approach for the development of new products through enhanced pathways offered by the agency, in particular providing the means for small institutions efficient means to meet requirements of the approval process. The field is growing rapidly – with three new approvals this past year in gene therapies and more than 500 active investigational new drug applications with the agency now. To facilitate rapid consideration he said that the agency will be issuing guidance documents, among which will be a pathway for accelerated approval endpoints for certain gene therapy products and that the first therapeutic category addressed will be for hemophilia.
- Congress Passes “Right to Try” – The House of Representatives passed the “Right to Try” legislation that provides an authorized pathway for patients with a terminal illness to seek direct access to drugs under investigation for safety and efficacy. Need for the legislation was perceived by many to be more political than practical given the already existing process of early access or compassionate use overseen by FDA. The new legislation removed federal oversight and Commissioner Gottlieb in prior Congressional testimony expressed reservations about it. The ultimate decision for expanded access under any pathway is with the sponsoring pharmaceutical company that is investigating the drug. The bill heads to the White House for signature to become law.
Things to Keep an Eye on This Week
- Not much going on that caught my eye. Congress is gone, no FDA Advisory Committee meetings.
Regulatory Developments in Pharma/Biotech/Devices
- Advisory Committee Votes Against Approval Recommendation for buprenorphine sublingual spray as treatment for moderate to severe pain
- New drug-eluding stent approved
- FDA Approves Treatment for PKU, a Rare Genetic Condition
- AI device approved to detect wrist fractures
Photograph by Lynne Bertram