UK Renews Puldysa Early Access Program for Duchenne Patients

Oral Puldysa (idebenone), Santhera Pharmaceuticals‘ investigational treatment for breathing problems caused by Duchenne muscular dystrophy (DMD), will be available to eligible patients in the U.K. for another year via the renewal of its Early Access to Medicines Scheme (EAMS), the company announced.

In agreeing to this renewal, the country’s Medicines and Healthcare products Regulatory Agency (MHRA) supported the positive scientific opinion it issued in 2017, when Puldysa was first brought into the EAMS for Duchenne patients in the U.K.

“This EAMS renewal for idebenone enables a much-needed therapeutic option for DMD patients with deteriorating respiratory function who have no real treatment alternative,” Kristina Sjöblom Nygren, MD, chief medical officer and head of development at Santhera, said in a press release. “We welcome the continued recognition by the UK’s MHRA of the positive benefit-risk of idebenone in this patient population.”

Puldysa contains idebenone, a synthetic small molecule that resembles the antioxidant ubiquinone (also known as coenzyme Q10), which is important for mitochondria, the so-called powerhouses of cells, to make energy.

The therapy is designed to improve mitochondrial function by increasing production of adenosine triphosphate — ATP, the “energy currency” of cells — and by reducing the production of reactive oxygen species, which are toxic free radicals that damage cellular structures.

Puldysa is being developed to treat respiratory symptoms (i.e., breathing difficulties) caused by DMD. It has been named a promising innovative medicine by U.K. health authorities, and granted rare pediatric disease and fast track designations by the U.S. Food and Drug Administration.

The EAMS covers Puldysa’s use by Duchenne patients, ages 10 and older, who are in active respiratory decline and not taking glucocorticoids, a class of corticosteroid. Patients previously treated with glucocorticoids, or in whom glucocorticoid treatment is not tolerated or considered inadvisable, may also be eligible.

At present, 84 Duchenne patients in the U.K. are using Puldysa under this early access program, the company stated in its release.

Santhera submitted a request to the European Medicines Agency for Puldysa approval to treat respiratory difficulties in people with DMD who are not on glucocorticoids last year. An opinion by the EMA’s Committee for Medicinal Products for Human Use is expected by late 2020.

The submission is supported by data from the Phase 3 clinical trial DELOS (NCT01027884). This study enrolled 64 people with DMD, ages 10 to 18, who were randomly assigned to Puldysa or a placebo three times daily for a year, without glucocorticoids.

Results indicated that Puldysa (then under the brand name Raxone) significantly slowed the decline of respiratory function, delaying the need for assisted ventilation by three years. In turn, results from the SYROS real-world study in 18 patients who completed the DELOS trial further supported the long-term benefits of Puldysa.

An ongoing and larger Phase 3 trial, called SIDEROS (NCT02814019), is assessing Puldysa in DMD patients, ages 10 and older, who are on a stable dose of glucocorticoids. If results are favorable, they are expected to support an approval request for this Duchenne patient group in both the EU and the U.S.