Rare Disease Roundup: FDA Panel Votes 9-3 Against a Duchenne Therapy, and 5 More

A rare and ultra-rare disease special, covering the last two weeks across all three of our categories -- clinical trials, deals, and regulatory action. An FDA advisory committee voted 9-3 against Capricor's deramiocel for Duchenne muscular dystrophy cardiomyopathy days after The Lancet published its positive Phase 3 data, Otsuka's VOYXACT became the first IgA nephropathy therapy to halt kidney decline, and the FDA granted orphan drug status to a gene therapy for an ultra-rare metabolic disorder affecting GABA metabolism. Six stories in total. Stories covered: - FDA panel votes 9-3 against Capricor's deramiocel, days after The Lancet published its Phase 3 data - Otsuka's VOYXACT becomes the first therapy to halt IgA nephropathy kidney decline - Beam Therapeutics doses first patient in its pivotal alpha-1 antitrypsin deficiency trial - Mironid raises $46M for a rare polycystic kidney disease drug - Galibra's gene therapy earns orphan drug and rare pediatric disease designations for SSADH deficiency - Regeneron's garetosmab awaits an FDA decision in fibrodysplasia ossificans progressiva The Clinical Brief summarizes publicly available regulatory and clinical trial data for informational purposes only. Not medical or investment advice. Always verify against primary sources -- full citations in the closing card below. Chapters: 00:00 Intro 00:13 FDA panel votes 9-3 against Capricor's deramiocel in DMD 01:11 VOYXACT becomes the first therapy to halt IgA nephropathy kidney decline 02:06 Beam doses first patient in its pivotal alpha-1 antitrypsin deficiency trial 02:50 Mironid raises $46M for a rare polycystic kidney disease drug 03:34 Galibra's gene therapy earns orphan and rare pediatric designations for SSADH deficiency 04:23 Regeneron's garetosmab awaits an FDA decision in fibrodysplasia ossificans progressiva 05:08 Wrap-up Stories & sources this week: 1. FDA panel votes 9-3 against Capricor's deramiocel in DMD https://www.globenewswire.com/news-release/2026/07/30/3336165/0/en/Capricor-Therapeutics-Provides-Update-on-FDA-Advisory-Committee-Meeting-for-Deramiocel.html https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)01385-1/fulltext https://www.biopharmadive.com/news/capricor-fda-vote-deramiocel-duchenne-cardiomyopathy/826465/ 2. VOYXACT becomes the first therapy to halt IgA nephropathy kidney decline https://www.otsuka-us.com/news/otsuka-unveils-unprecedented-phase-3-visionary-two-year-egfr-results-demonstrating-voyxactr https://www.otsuka.co.jp/en/company/newsreleases/2026/20260804_2.html 3. Beam doses first patient in its pivotal alpha-1 antitrypsin deficiency trial https://www.globenewswire.com/news-release/2026/08/04/3338130/0/en/Beam-Therapeutics-Reports-Second-Quarter-2026-Financial-Results-and-Announces-First-Patient-Dosed-in-Global-Pivotal-Cohort-of-BEAM-302-Trial-in-Alpha-1-Antitrypsin-Deficiency-AATD.html 4. Mironid raises $46M for a rare polycystic kidney disease drug https://www.globenewswire.com/news-release/2026/08/05/3338892/0/en/Mironid-raises-46-million-in-Series-B-financing-to-advance-clinical-development-of-its-treatment-for-rare-kidney-disease.html https://www.pharmaceutical-technology.com/news/mironid-46m-series-b-progress-kidney-disease/ 5. Galibra's gene therapy earns orphan and rare pediatric designations for SSADH deficiency https://www.prnewswire.com/news-releases/galibra-neuroscience-receives-fda-orphan-drug-and-rare-pediatric-disease-designations-for-ssadh-deficiency-gene-therapy-302844393.html https://www.biospace.com/press-releases/galibra-neuroscience-receives-fda-orphan-drug-and-rare-pediatric-disease-designations-for-ssadh-deficiency-gene-therapy 6. Regeneron's garetosmab awaits an FDA decision in fibrodysplasia ossificans progressiva https://investor.regeneron.com/news-releases/news-release-details/garetosmab-biologics-license-application-accepted-fda-priority https://www.globenewswire.com/news-release/2026/02/19/3240927/0/en/Garetosmab-Biologics-License-Application-Accepted-for-FDA-Priority-Review-for-the-Treatment-of-Fibrodysplasia-Ossificans-Progressiva-FOP.html Subscribe for next week's roundup - sourced, fact-checked biotech and pharma news, three times a week. Daily updates in shorter form on Instagram: https://www.instagram.com/theclinicalbrief_newsupdates/