Duchenne Muscular Dystrophy (DMD) Patients Have Choice of Medications

DMD is an inherited progressive neurological disorder that primarily affects boys because it is located on the X chromosome (male gene). It is caused by genetic mutations that prevent the production of dystrophin, a key protein for supporting muscle health.

Symptoms appear as early as two years of age and typically progresses to cardiac and respiratory muscle problems beginning in the patient’s teenage years, leading to serious, life-threatening complications.

The FDA recently granted accelerated approval to Viltepso (Viltepso) injection from NS Pharma for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

This is the second FDA-approved targeted treatment for patients with this type of mutation. Approximately 8% of patients with DMD have a mutation that is amenable to exon 53 skipping.

Option Care Health, one of the nation’s largest independent provider of home and alternate site infusion services, was selected to participate in the limited distribution network of VILTEPSO™ (viltolarsen).

Viltepso is among a growing number of chronic infusion therapies for neuromuscular disorders that Option Care Health is able to deliver to patients at home or in one of the company’s more than 125 ambulatory infusion suites across the country.

The FDA previously in December 2019 granted accelerated approval to Vyondys 53 (golodirsen) injection to treat Duchenne muscular dystrophy (DMD) patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.

Orsini Pharmaceutical Services (Orsini) was selected to provide Sarepta Therapeutics’ approved therapy Vyondys 53 (golodirsen). Orsini also distributes Sarepta’s other DMD therapy, Exondys 51 (eteplirsen).


Takeaway: Orsini and Option Care Health provide specialty pharmacy expertise for complex neuromuscular conditions, with broad access to insurance networks and national service