aTyr Pharma’s Facioscapulohumeral Muscular Dystrophy Drug Granted Orphan Drug Status in EU

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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atyr pharmaBiotherapeutics company aTyr Pharma, Inc. was recently awarded orphan drug designation from the European Commission (EC) for its Resolaris therapy, designed to treat facioscapulohumeral muscular dystrophy (FSHD). The investigational drug is the company’s first Physiocrine-based therapy, and is currently being developed to become a first-in-class intravenous protein treatment for the disease.

The EMA orphan drug status was awarded to Resolaris to enable the acceleration of the drug’s development process. The designation is granted to methods of diagnosing, treating or preventing life-threatening or chronically debilitating diseases affecting less than 10,000 patients within the European Union.

“Our mission is to develop medicines that will make a meaningful difference to patients impacted by debilitating rare diseases,” stated the CEO and executive chairman of aTyr Pharma, John Mendlein, PhD. “We are very pleased that the EMA has recognized the potential of Resolaris™ for patients suffering from FSHD. The Agency’s decision is an important advancement for our promising Physiocrine-based medicines, and marks a key milestone in our strategy to deliver Resolaris to address the unmet needs of patients with this rare disease.”

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The designation was granted after the drug received a positive opinion from the Committee for Orphan Medicinal Products (COMP), making it eligible for several incentives such as development support, reduced fees on upcoming applications for marketing-authorization and extended market exclusivity when the drug receives approval.

aTyr Pharma is developing Resolaris to treat adults who suffer from FSHD, a rare and severe genetic myopathy for which there is currently no cure or approved treatment. The therapy contains an immune component distinguished by its extensive immune cell involvement, and is based on naturally released protein from the human skeletal muscle cells.

aTyr also recently announced the enrollment of the first patients in its 1b/2 double blind randomized place-controlled study that will examine the efficacy, safety, tolerability, pharmacokinetics and the activity of the new biological drug in the treatment of FSHD. The company aims to conduct the study on a total of 44 patients with FSHD from various clinical settings within the European Union. This is the first study that aTyr Pharm is conducting with an Physiocrine-based drug.

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