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This issue covers September 28, 2026 to October 4, 2026 and was published October 5, 2026. Read the latest week

The week in rare disease

Every FDA decision, trial change and new guide in rare disease, recorded day by day and published each Monday.

to · Issue 24

Camzyos and Emcitate approved, and 249 trial changes across 70 conditions

In the week of Sep 28th we recorded 249 changes to rare disease trials on ClinicalTrials.gov, 2 FDA decisions, and no trial that opened to new patients.

By Jason Hunter · Published Monday, , covering the week that ended the day before

Between September 28 and October 4, 2026, Trial Friend recorded 249 changes to rare disease trials on ClinicalTrials.gov across 70 conditions, including 30 that stopped enrolling. The FDA made 2 decisions on a rare disease treatment that week, all approvals: Approved, Camzyos for Obstructive hypertrophic cardiomyopathy, adolescents; Approved, Emcitate for MCT8 deficiency. Trial Friend published 9 new guides.

  1. SEP28Mon · 2026ApprovalEmcitate, plus 4 changes
  2. SEP29Tue · 202619trial changes, 1 guide
  3. SEP30Wed · 2026ApprovalCamzyos, plus 66 changes
  4. OCT1Thu · 202635trial changes, 2 guides
  5. OCT2Fri · 202657trial changes, 2 guides
  6. OCT3Sat · 202640trial changes
  7. OCT4Sun · 202628trial changes, 1 guide
FDA and moneyTrial changesNew guidesGreen tiles mark an approval or a trial opening, grey a refusal or a trial closing, an outline something still to come

Trials that opened and closed, September 28 to October 4

Status changes recorded on ClinicalTrials.gov this week, 31 in all

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How this issue is made

Every trial change comes from ClinicalTrials.gov, read twice a day for the conditions Trial Friend follows, and every FDA decision, designation and label change from the FDA's own records, Drugs@FDA and openFDA, read daily. Each line links to the page that holds its source. The issue is assembled on the Monday after the week and does not change afterwards, except to correct an error, which is noted on the page. Counts cover only the rare conditions on this site, so they are a floor for rare disease as a whole, not a total. Nothing here is medical advice.

Past weeks

Every issue stays here at its own address. The archive begins with the first week we recorded, the week of April 20th, 2026.

  1. #28Week of October 26thVoceryq is approved for RRP, three myasthenia gravis trials close to new patients, and Kite's CAR-T reaches past myeloma1 3
  2. #27Week of October 19thUltomiris approved for IgA nephropathy, 7 new guides1 7
  3. #26Week of October 12thEnspryng approved for Thyroid eye disease, 10 new guides1 10
  4. #25Week of October 5th5 new guides5
  5. #24Week of September 28thCamzyos approved for Obstructive hypertrophic cardiomyopathy, adolescents, Emcitate approved for MCT8 deficiency, 9 new guides2 9
  6. #23Week of September 21stAtebrioz approved for Fibrodysplasia ossificans progressiva, Gazyva approved for Idiopathic nephrotic syndrome, 9 new guides3 9
  7. #22Week of September 14thAqneursa approved for Ataxia-telangiectasia, Fayuvi approved for Sanfilippo syndrome type A, 9 new guides2 9
  8. #21Week of September 7thIsembyld approved for Spinal muscular atrophy, Joenja approved for Activated PI3K delta syndrome (APDS), children 4 to 11, 5 new guides2 5
  9. #20Week of August 31stZanvastro approved for Alexander disease1
  10. #19Week of August 24thBesremi approved for Essential thrombocythemia, Mimrylo approved for Polycythemia vera, 1 new guide4 1
  11. #18Week of August 17thGenglycos approved for Glycogen storage disease type 1a, Pasatru approved for Fibrodysplasia ossificans progressiva2
  12. #17Week of August 10thTrial changes across the conditions we follow
  13. #16Week of August 3rdOrzeyful approved for Narcolepsy type 11
  14. #15Week of July 27thTrial changes across the conditions we follow
  15. #14Week of July 20thTrial changes across the conditions we follow
  16. #13Week of July 13thFabhalta approved for IgA nephropathy1
  17. #12Week of July 6thTrutakna approved for IgA nephropathy1
  18. #11Week of June 29thTrial changes across the conditions we follow
  19. #10Week of June 22ndTrial changes across the conditions we follow
  20. #9Week of June 15thTrial changes across the conditions we follow
  21. #8Week of June 8thTrial changes across the conditions we follow
  22. #7Week of June 1stTrial changes across the conditions we follow
  23. #6Week of May 25th1 new guide1
  24. #5Week of May 18th1 new guide1
  25. #4Week of May 11th1 new guide1
  26. #3Week of May 4th4 new guides4
  27. #2Week of April 27th4 new guides4
  28. #1Week of April 20th2 new guides2