The Canadian charity CureSPG50 is pleased to announce that the  U.S Food and Drug Administration (FDA) has awarded Rare Paediatric Disease Designation (RPDD) to Melpida an investigation treatment for SPG50

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CureSPG50 is developing Melpida which seeks to slow the progression of the inherited neurodegenerative condition Spastic Paraplegia Type 50 also know as SPG50. SPG50 causes children affected by this terrible disease to become completely paralyzed with limited brain function.

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“We are extremely pleased to have obtained Rare Paediatric Disease Designation (RPDD),” said Terry Pirovolakis, who along with his wife Georgia started CureSPG50 after their son Michael was diagnosed with the rare genetic disease as an infant. 

 

“I would like to thank our incredible team for getting us this far, but we still have a long way to go. Every day is a day we can never get back and our children deserve to live better lives. "We will continue to move forward until all the funds are raised and the treatment is approved." said Pirovolakis. 

 

Melpida, which is a combination of two words – Michael and the Greek word for hope, “Elpida” – is what the Pirovolakis family started to represent Michael’s hopeful journey for a cure. Melpida is a one-time Gene Replacement therapy that replaces the defective gene with a healthy new one.

 

The clinical study will compare Melpida’s effectiveness in pediatric children, to the natural progression of SPG50 as documented in previous groups of patients.  CureSPG50 is still in talks to determine who will be the lead investigation center for the trial.

 

Rare Pediatric Disease (RPD) Designation is given to therapies with the potential to treat rare diseases; Rare Pediatric Disease Designation is granted to potential treatments for serious rare diseases that mainly affect children. A rare disease is defined as one that affects fewer than 200,000 people in the U.S.

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If at the time of submitting evidence of safety and efficacy to the FDA the rare disease designation is still applicable, the submission will qualify for Priority review and a rare disease voucher will be granted upon approval.  This voucher can be used for a priority review of a different treatment at a future point.

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“This designation will hopefully help get this life changing drug to our children faster,” Pirovolakis said.

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