Makindus, Inc. a specialty pharmaceutical development company focused on ophthalmology announced today that the European Medicines Agency (EMA) has granted orphan drug designation for their lead candidate, MI-100, for the treatment of Stargardt's Disease.
"We are extremely pleased to now have orphan drug designation for MI-100 to treat Stargardt's disease in place for Europe. This complements and reinforces our development program in the United States for which orphan designation has already been granted," stated Manohar Katakam, President and CEO of Makindus.
MI-100 is a novel formulation of a legacy compound that has been shown to have a clinical effect in improving visual acuity for Stargardt's patients in an investigator-initiated clinical trial. Makindus has developed MI-100 in a new sterile and more convenient ophthalmic dosage form. The proprietary packaging for MI-100 is a single bottle closed container mixing system that allows for a ready-to-use multi-dose eye dropper for easy administration.
Stargardt's disease, also known as juvenile macular degeneration, is a genetic disorder that causes progressive vision loss. The disorder affects a small area near the center of the retina called the macula which is responsible for sharp central vision needed for detailed tasks such as reading, driving, and recognizing faces. The signs and symptoms of Stargardt's disease typically appear in late childhood to early adulthood and worsen over time. The estimated prevalence in the U.S. and Europe is approximately 1 in 10,000 individuals. There is currently no cure or treatments.
Makindus recently met with the Food & Drug Administration (FDA) to review a clinical development program to evaluate MI-100 in a Phase 3 program. "We had a very productive meeting with the FDA and plan to start our Phase 3 trials by the end of this year," stated David Eveleth, PhD and Clinical Head for Makindus. "Stargardt's patients present a unique challenge for retinal specialists and we are looking forward to conducting the largest clinical trial that has ever been done in this orphan indication."
In obtaining orphan designation from the European Medicines Agency's Committee for Orphan Medicinal Products (COMP), Makindus will benefit from a number of incentives, including protocol assistance, a type of scientific advice specific for designated orphan medicines, and market exclusivity for ten years once the medicine is on the market.
Makindus is in the process of completing the clinical trial design and contracting for clinical supplies in order to enroll Stargardt's patients into a clinical trial by the end of the year. The company is currently in discussions with corporate partners to help fund and collaborate on the development of MI-100 for Stargardt's disease.