Drugs in Development with Regulatory flexibility

Table with 4 columns and 12 rows.
Rocket PharmaceuticalsDanon Disease - Highly Fatal Cardiomyopathy in kidsPhase 2 pivotal trial for acc approval completed enrollmentExpect completion late next year. Data on epidemiology earlier 25. Single arm trial, comparison to natural history. Outcomes are 12 months for LVMI and LAMP2 (biomarkers)
Capricor TherapeuticsDuchenne Muscular Dystrophy - Progressive disease in young kidsFiling for Full approval for DMD - CardiomyopathyExpect to file early next year and approval late next year. The original trial failed, but the FDA came to them after seeing OLE data.
Denali TherapeuticsHunter syndrome - Progressive disease in childrenPhase ½ study completePlans to file for AA in early 2025. Approval based on surrogate. Heparin sulfate is well established. High patient advocacy
SolenoPrader Willi syndrome - No approved therapies. People have uncontrollable urge to eatNear registrationalPDUFA Date March 27
Stealth medicineBard Syndrome - No approved therapies, KidsPDUFA Jan 29Advisory committee voted for approval after trial failed
Lexeo TherapeuticsFridrich’s ataxia Cardiomyopathy - Major cause of death, usually late middle agePhase 1 in progressPlans to initialize trial 2025. 10% LVMI reduction, FXN protein expression via IHC
Regulus TherapeuticsADPKD - Progressive disease requiring transplant (usually middle age)Phase 1 trial completing, Phase 2 starting next year after alignmentMeeting with FDA to determine path forward in Q4. Potential for AA based on cyst volume which was not allowed for Tolvaptan (other approved drug)
Disc MedicineErythropoeitic protoporphyria (patients can’t be in the sun at all)Completed Phase 2 trialMeet with FDA Q1 and update after. Positive on PPIX benefit but not secondary endpoint of time in sunlight. Use PPIX as biomarker for accelerated approval
UniqureHuntington’s disease - progressive neurological deficits and death in middle agePhase 2 completePotential AA based on small trial compared to natural history
Edgewise therapeuticsBecker Muscular Dystrophy - progressive muscle dysfunction in middle agePhase 2 trial completeTrends in NSAA, positive CK benefit. Unclear. Plans to go to the FDA
Ovid Therapeutics/TakedaDravet SyndromeMissed primary endpointTakeda plans to engage the FDA.
Larimar TherapeuticsFriedrich's AtaxiaFiling for approval late 2025Concerns over safety, approval based on Surrogate endpoint.