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Press Release

Option Care Selected to Provide Exondys 51™, First Approved Treatment for Most Common Form of Muscular Dystrophy

Option Care
Posted on: 21 Sep 16

Option Care Enterprises, Inc., one of the nation’s leading providers of home and alternate treatment site infusion services, has been selected as a contracted provider of the first drug therapy specifically for the treatment of Duchenne Muscular Dystrophy (DMD). Exondys 51™ (eteplirsen) injection, manufactured by Sarepta Therapeutics, is the first U.S. Food and Drug Administration-approved drug therapy for DMD, a debilitating and ultimately fatal inherited disorder that primarily affects boys.

Exondys 51™ is among the growing number of orphan therapies that Option Care has been chosen to provide to patients with complex conditions because of its signature Home Infusion Plus services, which feature high-touch, high-quality clinical management of infusion medicine, nursing support and care coordination.

“As the vanguard for the industry, Option Care is pioneering innovation in home infusion and forging partnerships with other health partners to build seamless transitions of care around patients,” said Paul Mastrapa, CEO of Option Care. “Our highly skilled nurses, dietitians and pharmacists are dedicated to providing compassionate, personalized care to patients and parents and supporting them as they cope with challenging illnesses.”

The most common fatal genetic disorder diagnosed in childhood, DMD is a progressive disease that causes loss of muscle, eventually affecting heart function and breathing: most patients are wheelchair-bound by age 12 and die in early adulthood. Because it is located on the X chromosome – the male gene – DMD primarily affects boys.

Exondys 51™ was approved by the FDA on September 19 for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. Because this form of DMD is a rare disorder and there was no approved treatment, Exondys 51™ was granted orphan product designation for fast track review. Prior to approval of Exondys 51™, treatment consisted of steroid therapy to prolong muscle function, assistive devices, and physical therapy.

Exondys 51™ was designed to provide therapeutic benefit to the largest subset of patients with DMD (13 percent), meaning about 2,000 U.S. patients would be candidates for the medication. In these patients a mutation on exon 51 of the dystrophin gene prevents the formation of dystrophin, a vital protein that strengthens muscle fibers and protects them from injury. Provided by intravenous (IV) infusion of 30 mg/kg once weekly, Exondys 51™ enables bypass of the mutation and creation of a truncated form of dystrophin.

The FDA approval was based on the surrogate endpoint of dystrophin increase in skeletal muscle observed in some Exondys 51™-treated patients. The FDA has concluded that the data submitted by the applicant demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in some patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping.

“Option Care continues to be selected by manufacturers seeking high-touch, high-skilled nursing and pharmacy support services for patients with rare diseases,” said Kendra Curry, PharmD, Corporate Director, Specialty Infusion Programs with Option Care. “They are impressed with our proven track record and appreciate that we work closely with the patient and physician to help ensure successful outcomes for those who require complex therapies.”

Option Care has a staff of more than 1,800 clinical experts, including pharmacists, more than 1,200 infusion nurses and more than 100 registered dietitians. This multidisciplinary team of specially trained clinicians provides individualized patient care plans, clinical monitoring of each infusion and comprehensive patient and caregiver education.

About Option Care

Option Care Enterprises, Inc. (Option Care) is one of the nation’s largest and most trusted providers of home and alternate treatment site infusion services. An industry leader, the company draws on nearly 40 years of clinical care experience to offer patient-centered therapy management. Option Care’s signature Home Infusion Plus services include the clinical management of infusion medicines, nursing support and care coordination. Option Care’s more than 1,800 clinicians – including pharmacists, nurses and dietitians – are able to provide home infusion service coverage for nearly all acute and chronic patients across the United States. Learn more at

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Business Wire

Last updated on: 21/09/2016

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