Ra Pharmaceuticals announced that they have received Orphan Drug Designation for RA101495, their investigational treatment for paroxysmal nocturnal hemoglobinuria (PNH).

RA101495 is a synthetic macrocyclic peptide inhibitor of complement component 5 (C5) and is currently being tested in a Phase 2 trial as a self-administered subcutaneous (SC) injection for the treatment of PNH. Present standard of care for PNH includes biweekly intravenous infusions.

“We have designed RA101495 for once-daily, subcutaneous self-administration, an approach which has the potential to ease this burden, improve convenience, and provide much-needed dosing flexibility,” said Doug Treco, PhD, President and CEO of Ra Pharma. “We are encouraged by our initial Phase 2 data, which showed near-complete inhibition of hemolysis and a favorable safety and tolerability profile.” 

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PNH is a rare and life-threatening blood disorder in which the complement system attacks and destroys red blood cells. Phase 1 studies found the treatment to be well tolerated in healthy volunteers and to demonstrate sustained and near complete suppression of hemolysis and complement activity. The treatment gained Orphan Drug Designation for PNH in Europe last Fall. 

The company is also developing RA101495 as a possible treatment for refractory generalized myasthenia gravis (rMG) and lupus nephritis (LN).

For more information visit RaPharma.com.