The European Commission (EC) has recently granted its Orphan Drug designation to AR101, a PKCβ inhibitor for the treatment of Ehlers-Danlos syndrome (EDS). This decision was based off of a positive recommendation from the Committee for Orphan Medicinal Products (COMP), which is part of the European Medicines Agency (EMA).

Orphan Drug Designation for AR101

In order for an investigational treatment to be considered for the Orphan Drug designation, it must meet the following criteria:

• Meant to treat a life-threatening or seriously debilitating condition
  • This condition must impact less than five of every 10,000 people in the European Union
• There is clinical or non-clinical data that suggests the medication could lead to clinically relevant outcomes

If a drug meets these criteria and is granted the designation, the drug developer is awarded with incentives and benefits. These include lower regulatory fees, assistance with clinical protocol, access to a centralized marketing authorization procedure, 10 years of market exclusivity, and differentiated evaluation procedures for Health Technology Assessments in certain countries.

AR101 has already received Orphan Drug designation in the US. The next step is to evaluate this drug’s safety and efficacy in the PREVEnt trial, which is planned to begin in the middle of 2022.

Find out more about this designation and AR101 here.

About EDS

Ehlers-Danlos syndrome (EDS) is a group of disorders that affect the connective tissue. There are numerous forms, all of which are defined by the symptoms they present. Possible effects include:

• Skin that is velvety, fragile, and highly elastic
• Easy bruising
• Poor healing
• “Cigarette paper” scars
• Flexible, unstable, and loose joints
  • These joints dislocate and sprain easily
• Muscle and tendon tears
• Deformities in the fingers, spine, and neck
• Digestive tract, respiratory system, and vessels of the brain malfunctions
• Internal bleeding
• Stroke
• Sudden vessel rupture
• Varicose veins
• Heart rhythm disorders
• Heart valve disease
• Pregnancy complications in females

These symptoms are the result of inherited genetic mutations that cause collagen and related proteins to become defective.