Pfizer's Duchenne Muscular Dystrophy Trial Setback Benefits Sarepta and Others | DelveInsight

Duchenne muscular dystrophy (DMD) is a genetic disorder that predominantly affects children, leading to progressive muscle weakening and degeneration. This condition is the most common form of muscular dystrophy in children and is one of nine distinct types. In the United States, there were approximately 17,000 prevalent cases of DMD in 2023, with the highest concentration in children aged 5-9 years. These numbers are expected to increase by 2034.

Comorbidities often accompany DMD, with scoliosis being the most prevalent, affecting around 20% of DMD patients. Other common comorbidities include ADHD, cardiomyopathy, and obsessive-compulsive disorders, which impacted approximately 19%, 18%, and 15% of patients, respectively, in 2023.

The standard treatment for DMD involves corticosteroids, such as prednisone and deflazacort, which have been used for over two decades to enhance muscular strength. Surgical interventions may also be required to address complications such as contractures, spinal curvature, or scoliosis, which can impact breathing and mobility. Cardiac devices like pacemakers may be used to support heart function.

Sarepta Therapeutics is a significant player in the DMD market, with three approved therapies and two more in the pipeline. Despite its dominance, the market is becoming increasingly competitive. Regulatory approvals for DMD treatments often show geographic variations, with some products being approved in one region but not in another, reflecting differing standards between regulatory bodies like the EMA and FDA.

The current US DMD treatment market includes several approved therapies such as EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), VILTEPSO (viltolarsen), and ELEVIDYS (delandistrogene moxeparvovec). Santhera Pharmaceuticals recently launched AGAMREE (vamorolone) in Germany for DMD patients aged four and above, marking its entry into the commercial biopharma sector.

ELEVIDYS, the first FDA-approved gene therapy for DMD, received accelerated approval in June 2023. This therapy is intended for ambulatory children aged 4-5 years with a confirmed dystrophin gene mutation. Patients are required to take oral corticosteroids before and after receiving ELEVIDYS and undergo weekly blood tests to monitor liver enzyme levels for three months post-treatment.

VYONDYS 53, developed by Sarepta Therapeutics, is approved under the accelerated approval pathway for treating DMD patients with a specific mutation of the DMD gene. This approval was based on observed increases in dystrophin production in patients' skeletal muscle. The FDA granted VYONDYS 53 New Chemical Entity exclusivity until December 2024 and Orphan Drug Exclusivity until December 2026.

The DMD market is highly competitive, with numerous companies such as FibroGen, Santhera Pharmaceutical, Italfarmaco, ReveraGen BioPharma, and others conducting clinical trials to enhance DMD treatment options. Promising drugs in development include Pizuglanstat (TAS-205), SRP-5051, CAP-1002 (Allogeneic Cardiosphere-derived Cells), ITF2357 (Givinostat), among others, which could significantly impact the DMD market.

Italfarmaco's Givinostat (ITF2357), an oral histone deacetylase inhibitor, is currently in Phase III trials. The FDA has granted it Orphan Drug, Rare Pediatric Disease, and Fast Track designations for DMD treatment. Additionally, TAS-205 (pizuglanstat) by Taiho Pharmaceutical, a selective inhibitor of hematopoietic prostaglandin D synthase, is in Phase III trials and shows promise for DMD treatment irrespective of the type of dystrophin gene mutation.

DelveInsight projects that the DMD market will grow from USD 2.1 billion in 2023 at a significant CAGR through 2034, driven by increased diagnosis, patient awareness, and a robust clinical pipeline. As new therapies gain regulatory approval, they are expected to transform the DMD treatment landscape, offering new care standards and fostering medical innovation and economic growth.