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The Post-Makary FDA Pivot: Reversals, Resubmissions, and a Return to Advisory Committees

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The Post-Makary FDA Pivot: Reversals, Resubmissions, and a Return to Advisory Committees

The U.S. Food and Drug Administration (FDA) continues to exhibit a profound operational and regulatory shift following the spring 2026 departures of former Commissioner Marty Makary and CBER Director Vinay Prasad. This "post-Makary pivot" is characterized by unprecedented regulatory flexibility, allowing companies to directly resubmit previously rejected applications and scheduling advisory committee reviews to resolve outstanding disputes.1

Saol Therapeutics Resubmits SL1009 (DCA)

On June 30, 2026, Saol Therapeutics became the latest biotech to leverage this regulatory shift by resubmitting its New Drug Application (NDA) for SL1009 (sodium dichloroacetate, DCA). The candidate is being developed for pyruvate dehydrogenase complex deficiency (PDCD), an ultra-rare, life-threatening mitochondrial disease with no approved therapies.

SL1009 was rejected in September 2025 under the previous FDA leadership, a decision that would have required "several years" and "significant financial resources" to resolve under standard procedures. Following productive Type A and Type C meetings, the FDA allowed Saol to resubmit with additional survival analyses. The new package includes 36 natural history-matched pairs (up from 28) and an additional 18 months of data demonstrating a 91% reduction in the risk of death with SL1009. CEO Dave Penake indicated that the company expects the FDA to accept the filing and set a PDUFA target action date prior to July 30, 2026.

Confirmed Advisory Committee Dates for Capricor and Replimune

The FDA’s Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) has officially scheduled back-to-back public meetings in late July 2026 to evaluate two other high-profile therapies that were previously rejected under the former leadership:

  1. Capricor's Deramiocel (July 29, 2026): The CTGTAC will meet at the FDA White Oak Campus (9:30 a.m. – 4:50 p.m. ET) to review the BLA for deramiocel (human allogeneic cardiosphere-derived cells) for the treatment of cardiomyopathy in Duchenne muscular dystrophy (DMD). The PDUFA target action date is scheduled for August 22, 2026.
  2. Replimune's RP1 (July 30, 2026): The CTGTAC will meet the following day to review the BLA for vusolimogene oderparepvec (RP1) in combination with nivolumab for patients with advanced melanoma who have previously progressed on anti-PD-1 therapy. The PDUFA target action date is August 2, 2026.

These back-to-back reviews underscore the agency's reliance on independent expert panels to rebuild consensus and navigate controversial, previously blocked applications.

  1. An instance of Leadership churn at regulatory agencies triggers a systemic softening of clinical standards. — The departure of conservative FDA leaders has triggered widespread regulatory flexibility, allowing developers to quickly resubmit previously blocked applications. ↩︎

Part of

This finding is an example of a pattern recurring across your work:

Revision history

  • Update the existing note with confirmed CTGTAC meeting details for Capricor and Replimune, and document Saol's SL1009 resubmission as a key post-Makary pivot event.
    · by the agent
  • Update the Post-Makary FDA Pivot finding with fresh details on the FDA's 2,200-person hiring initiative, CBER and CDER acting leadership, and Replimune's third BLA resubmission and upcoming late-July advisory committee meeting.
    · by the agent
  • Update with Saol Therapeutics' SL1009 resubmission on June 30, 2026, and details on the upcoming July CTGT Advisory Committee meetings for Capricor's deramiocel (July 29) and Replimune's RP1 (July 30) along with the post-Makary FDA leadership vacuum.
    · by the agent
  • Update the Post-Makary FDA Pivot note to include Saol Therapeutics' June 30, 2026 resubmission of SL1009 (DCA) for PDCD, illustrating the wider pattern of rapid biotech regulatory turnarounds.
    · by the agent
  • Update the existing post-Makary FDA pivot note with the newly confirmed late-July 2026 Advisory Committee dates for Capricor's deramiocel and Replimune's RP1.
    · by the agent
  • Update the post-Makary FDA pivot with the June 26 BLA resubmission acceptance for Replimune's RP1, Sobi's manufacturing-related CRL, and upcoming Q3 2026 AdCom milestones.
    · by the agent
  • Update the post-Makary FDA pivot with the June 26 BLA resubmission acceptance for Replimune's RP1, Sobi's manufacturing-related CRL, and upcoming Q3 2026 AdCom milestones.
    · by the agent
  • Update the post-Makary FDA pivot with the June 26 BLA resubmission acceptance for Replimune's RP1, Sobi's manufacturing-related CRL, and upcoming Q3 2026 AdCom milestones.
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  • Update note on FDA regulatory pivot to include Sarepta's sNDA acceptance, uniQure's BLA pathway, and upcoming Advisory Committees.
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  • Update the FDA regulatory pivot note with the acceptance of Capricor's and Replimune's BLA resubmissions, their scheduled advisory committee meetings, and the resignation of acting OTP director Vijay Kumar.
    · by the agent
  • Update the Post-Makary FDA Pivot note with the dual announcements on June 26, 2026: Capricor's deramiocel scheduled for a July 29 AdCom and August 22 PDUFA, and Replimune's RP1 BLA accepted as a Class 1 response with an August 2 PDUFA and late-July AdCom.
    · by the agent
  • Update with the official scheduling of AdComs for Capricor's deramiocel (July 29, 2026) and Replimune's RP1 (late July 2026) and the broader policy context under Acting FDA Commissioner Kyle Diamantas.
    · by the agent
  • Update with the official scheduling of AdComs for Capricor's deramiocel (July 29, 2026) and Replimune's RP1 (late July 2026) and the broader policy context under Acting FDA Commissioner Kyle Diamantas.
    · by the agent
  • Update with the official scheduling of AdComs for Capricor's deramiocel (July 29, 2026) and Replimune's RP1 (late July 2026) and the broader policy context under Acting FDA Commissioner Kyle Diamantas.
    · by the agent
  • Synthesized the regulatory shift at the FDA post-Makary and Prasad, using the June 26, 2026 announcements of scheduled adcomms for Capricor's deramiocel and Replimune's RP1.
    · by the agent
  • Synthesized the regulatory shift at the FDA post-Makary and Prasad, using the June 26, 2026 announcements of scheduled adcomms for Capricor's deramiocel and Replimune's RP1.
    · by the agent
  • Synthesized the regulatory shift at the FDA post-Makary and Prasad, using the June 26, 2026 announcements of scheduled adcomms for Capricor's deramiocel and Replimune's RP1.
    · by the agent
  • Synthesized the regulatory shift at the FDA post-Makary and Prasad, using the June 26, 2026 announcements of scheduled adcomms for Capricor's deramiocel and Replimune's RP1.
    · by the agent
  • Synthesized the regulatory shift at the FDA post-Makary and Prasad, using the June 26, 2026 announcements of scheduled adcomms for Capricor's deramiocel and Replimune's RP1.
    · by the agent