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The week in rare disease

to · Issue 28 · The latest week

Voceryq is approved for RRP, three myasthenia gravis trials close to new patients, and Kite's CAR-T reaches past myeloma

The FDA approved Voceryq on Thursday, 14 months after Papzimeos, and families with recurrent respiratory papillomatosis now have two treatments to ask about. Quieter news mattered too. Three myasthenia gravis trials stopped taking new patients on the same day, and 223 trial changes made it the busiest week since late September.

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

  1. OCT26Mon18trial changes
  2. OCT27Tue31trial changes
  3. OCT28Wed21trial changes, 1 guide
  4. OCT29Thu71trial changes, 1 guide
  5. OCT30FriApprovalVoceryq, plus 19 changes
  6. OCT31Sat61trial changes
  7. NOV1Sun2trial changes
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

Three storylines

Each thread carries on from earlier weeks and picks up again next week. Every line links to its page.

RRP gets its second treatment in 14 months

Recurrent respiratory papillomatosis

Until August 2025 the only treatment for RRP was repeat surgery. Voceryq joins Papzimeos, so the question for families moves from whether there is anything to which one fits.

  1. FDA decisionApproved. Voceryq (INO-3107) for adults with recurrent respiratory papillomatosis, an accelerated approval that INOVIO must confirm in further trials.FDA
  2. FDAThe FDA called it the first plasmid DNA immunotherapy approved in the US. In the 32-patient trial, 7 of 26 people who had 3 or more surgeries the year before needed none the year after.FDA
  3. EarlierOur explainer on the INO-3107 data, how the injections work and how it compares with Papzimeos.Trial Friend
  4. What to watchINOVIO expects Voceryq to be available to order in about 60 days, with a full launch in the first quarter of 2027.Q1 2027
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Three myasthenia gravis trials close on one day

Myasthenia gravis

Three trials stopped enrolling on October 29th, which usually means they filled rather than failed. With gefurulimab's FDA decision pending, the field is shifting from recruiting to waiting for results.

  1. Trial statusNo longer enrolling. Vertex's Phase 2 study of povetacicept in generalized myasthenia gravis, NCT07501702, which had added sites in Tampa and Chapel Hill the same day.ClinicalTrials.gov
  2. Trial statusNo longer enrolling. argenx's efgartigimod study NCT04833894 and UCB's Phase 4 rozanolixizumab study NCT07246564 closed to new patients.ClinicalTrials.gov
  3. Trial sitesDianthus's Phase 3 study of claseprubart, NCT07647510, went from 4 sites to 12, all 8 new ones in Georgia, the country.ClinicalTrials.gov
  4. What to watchGefurulimab's FDA decision date has not been disclosed. We email the day it is.Not set
See the myasthenia gravis trials recruiting now →

Kite's CAR-T reaches past myeloma

Multiple myeloma

Anito-cel faces its myeloma decision on December 23rd. The same cell therapy is now being tested in people whose plasma cells drive autoimmune disease, and that trial grew this week.

  1. Trial sitesKite's Phase 1 study of anitocabtagene autoleucel in non-cancer plasma cell diseases, NCT06626919, added 8 sites, including Los Angeles, Orange and Palo Alto.ClinicalTrials.gov
  2. Who can joinThe same study updated who can join. Adults 18 and older with a qualifying condition, so check the page again if you were turned away.ClinicalTrials.gov
  3. New guideMyeloma's third CAR-T, anito-cel, faces a December 23rd FDA decision next to Carvykti and Abecma.Trial Friend
  4. What to watchThe FDA is due to decide on anito-cel for relapsed or refractory multiple myeloma.
Compare the myeloma CAR-T and bispecific options →

Trials that opened and closed

Status changes recorded on ClinicalTrials.gov this week, 28 in all

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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