Rare Disease Trial Updates: April 2026

In April 2026, Trial Friend tracked 136 rare disease clinical trial updates across 53 conditions, including 11 FDA-related actions and 16 trial status changes, alongside enrollment updates and new trial site openings sourced from ClinicalTrials.gov and FDA announcements.

This is a permanent monthly archive. For the latest updates, see the live current events feed.

Major Updates: FDA Actions, Status Changes & Results

FDA ApprovalApr 30

FDA Approves First Non-Antipsychotic Drug to Treat Agitation Associated with Dementia

The U.S. Food and Drug Administration today approved an expanded use for Auvelity (dextromethorphan hydrobromide and bupropion hydrochloride) extended-release tablets to treat agitation associated with dementia due to Alzheimer’s disease in adults.

Status ChangeEssential ThrombocythemiaApr 30Swedish Orphan BiovitrumNCT03165734

No longer enrolling new participants

FDA NewsApr 30

FDA Proposes to Exclude Semaglutide, Tirzepatide, and Liraglutide on 503B Bulks List

FDA is proposing to exclude semaglutide, tirzepatide, and liraglutide from the 503B bulks list, finding no clinical need for outsourcing facilities to compound these drugs from bulk substances.

Status ChangeEpidermolysis BullosaApr 30TWi Biotechnology, Inc.NCT06073132

No longer enrolling new participants

Status ChangeEosinophilic Granulomatosis with PolyangiitisApr 30Imperial College LondonNCT06230354

No longer enrolling new participants

Status ChangeEosinophilic EsophagitisApr 30University of North Carolina, Chapel HillNCT06352073

No longer enrolling new participants

Status ChangeAmyotrophic Lateral SclerosisApr 29ProJenXNCT05279755

No longer enrolling new participants

FDA NewsApr 28

FDA Announces Major Steps to Implement Real-Time Clinical Trials

The U.S. Food and Drug Administration today announced two major steps as part of an initiative to advance the implementation of real-time clinical trials (RTCT).

Status ChangeUrea Cycle DisordersApr 28Baylor College of MedicineNCT04612764

No longer enrolling new participants

Status ChangeSarcoidosisApr 27Assistance Publique - Hôpitaux de ParisNCT05689879

No longer enrolling new participants

FDA NewsApr 24

FDA Accelerates Action on Treatments for Serious Mental Illness Following Executive Order

On April 18, President Trump issued an Executive Order directing the U.S. Department of Health and Human Services (HHS) to accelerate access to treatments for patients with serious mental illness, including devastating, complex, and treatment-resistant conditions.

Status ChangeHemophilia AApr 24CSL BehringNCT06738485

No longer enrolling new participants

Status ChangeGraves’ DiseaseApr 24Tourmaline Bio, Inc., a Novartis CompanyNCT06088979

No longer enrolling new participants

FDA ApprovalApr 23

FDA Approves First-Ever Gene Therapy for Treatment of Genetic Hearing Loss Under National Priority Voucher Program

The U.S. Food and Drug Administration today approved Otarmeni (lunsotogene parvec-cwha), the first-ever dual adeno-associated virus (AAV) vector-based gene therapy.

Status ChangeSickle Cell DiseaseApr 23Agios Pharmaceuticals, Inc.NCT06924970

No longer enrolling new participants

Status ChangeMultiple System AtrophyApr 23NS Pharma, Inc.NCT06053814

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophyApr 23NS Pharma, Inc.NCT06053814

No longer enrolling new participants

Status ChangeDuchenne Muscular DystrophyApr 23NS Pharma, Inc.NCT06053814

No longer enrolling new participants

Status ChangeIdiopathic Pulmonary FibrosisApr 23Vicore Pharma ABNCT06588686

No longer enrolling new participants

Status ChangeBecker Muscular DystrophyApr 23NS Pharma, Inc.NCT06053814

No longer enrolling new participants

FDA NewsApr 23

CMS and FDA Announce RAPID Coverage Pathway to Accelerate Patient Access to Life-Changing Medical Devices

CMS and FDA announce the Regulatory Alignment for Predictable and Immediate Device (RAPID) coverage pathway, a new pathway designed to expedite access to certain FDA-designated Class II and Class III Breakthrough Devices for people with Medicare.

FDA NewsApr 20

FDA Achieves Year 1 Goals in Reducing Animal Testing in Drug Development

Since publishing roadmap last April, agency has successfully launched several key initiatives to replace animal testing with better alternatives

Status ChangeApr 19Vera Therapeutics, Inc.NCT07020923

No longer enrolling new participants

FDA ApprovalApr 16

FDA Takes Step Forward on Testosterone Therapy for Men

FDA is taking an initial step to advance treatment options for men’s health by encouraging sponsors of approved testosterone replacement therapy products to contact FDA for information if they are interested in pursuing a potential new indication for low libido in men with idiopathic hypogonadism.

FDA ApprovalApr 14

FDA Issues Draft Guidance on Genome Editing Safety Standards to Advance Gene Therapy Development

The U.S. Food and Drug Administration today issued a draft guidance for sponsors seeking approval of human gene therapy products involving genome editing technologies.

FDA NewsApr 13

FDA Reminds More Than 2,200 Sponsors and Researchers to Disclose Trial Results

The U.S. Food and Drug Administration has reminded more than 2,200 medical product companies and researchers of the requirements to submit certain clinical trial results information to ClinicalTrials.gov.

FDA ApprovalApr 1

FDA Approves First New Molecular Entity Under National Priority Voucher Program

The U.S. Food and Drug Administration today approved Foundayo (orforglipron) marking the fifth approval under the Commissioner's National Priority Voucher (CNPV) pilot program.

Trial Site & Enrollment Activity

sites_addedEssential ThrombocythemiaApr 30Novartis PharmaceuticalsNCT07357727

Added 1 new site: Seoul, South Korea

sites_addedCongenital Adrenal HyperplasiaApr 30Crinetics Pharmaceuticals Inc.NCT07159841

Added 8 new sites: Córdoba, Argentina, Buenos Aires, Argentina, Edegem, Belgium and 5 more

sites_addedCongenital Adrenal HyperplasiaApr 30Crinetics Pharmaceuticals Inc.NCT07144163

Added 8 new sites: Rochester, Minnesota, CABA, Argentina, Curitiba, Brazil and 5 more

sites_addedEssential ThrombocythemiaApr 30National Cancer Institute (NCI)NCT06661915

Added 4 new sites: Palo Alto, California, Charlotte, North Carolina, Concord, North Carolina and 1 more

sites_removedEpidermolysis BullosaApr 30TWi Biotechnology, Inc.NCT06073132

Reduced from 36 to 30 sites

Enrollment UpdateEosinophilic Granulomatosis with PolyangiitisApr 30Imperial College LondonNCT06230354

Target enrollment decreased from 66 to 42

eligibility_changeEosinophilic Granulomatosis with PolyangiitisApr 30Imperial College LondonNCT06230354