EMA grants orphan drug designation for PNH to Amyndas Pharmaceuticals’ novel complement inhibitor
Amyndas Pharmaceuticals SA, the complement therapeutics company, announced that the European Medicines Agency (EMA) has granted AMY-101, a potent complement C3 inhibitor, an Orphan Drug Designation for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
PHILADELPHIA - Aug. 27, 2014 - PRLog -- “Receiving the Orphan Drug Designation for PNH speaks to the need for new treatment options for patients with PNH” said Dr. Lambris, Founder of Amyndas and Professor of Research Medicine in the Department of Pathology and Laboratory Medicine at the University of Pennsylvania. “In PNH, it is clear that about 30 percent of the patients are not optimally responding to therapy; when current treatment fails, clinicians have limited options to offer these patients. With its ability to inhibit complement centrally, at the level of C3 instead of C5, the novel peptide drug AMY-101 could increase the number of PNH therapy responders and benefit many more patients suffering from PNH”.
“We believe that AMY-101 could represent a significant therapeutic advantage over treatments currently available for PNH” said Dr. Lambris. “Additionally, since AMY-101 binds tightly to C3 with a long half-life, the drug could be self-administered subcutaneously every 12 hours, giving patients better control and dramatically reducing the need for clinic visits.”
“This is an important milestone for AMY-101 and brings Amyndas one step closer to making this novel compound available to PNH patients,” said Dr. Lambris.
The Orphan Drug Designation may qualify Amyndas for a number of benefits under the EU Regulation on orphan medicinal products (Regulation (EC) No 141/2000). These benefits include assistance in study design from the EMA, fee reductions, eligibility for orphan disease development grants, significant tax incentives and potential for expedited drug development and a ten-year period of orphan drug exclusivity in Europe upon product approval. Amyndas is planning to move AMY-101 into the clinic for first-in-human trials in 2015, and the Orphan Drug Designation should prove helpful in the clinical development of this drug candidate.
AMY-101 is a novel therapeutic based on the second-generation compstatin analogue Cp40, discovered by Dr. John Lambris of the University of Pennsylvania, a leading researcher in the field of complement and the founder of Amyndas Pharmaceuticals. AMY-101 inhibits complement component C3 and interrupts the complement activation cascade, which plays a pivotal role in PNH. Compared to previous compstatin analogues, AMY-101 shows 6,000-fold improvements in binding affinity to human C3, enhanced inhibitory potency and an extended circulating half-life in vivo.
Amyndas has an exclusive, worldwide licensing agreement with the University of Pennsylvania that provides broad rights to develop and commercialize University of Pennsylvania’
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare but life-threatening hematological disorder. In PNH, important regulatory proteins are missing from the surface of blood cells, leaving them vulnerable to complement attack. Activation of complement can cause red blood cells to break apart, a process called hemolysis, which clinically results in severe anemia and contributes to a high risk of clotting.
By targeting complement component C5, the monoclonal antibody eculizumab (Soliris®, an Alexion Pharmaceuticals drug) - to date the only approved therapeutic for PNH – reduces hemolysis and the need for blood transfusions for many PNH patients. However, one third of PNH patients continue to require blood transfusions to manage their anemia; this insufficient response is due to fragments of complement C3 proteins on the surface of their red blood cells, which are eventually attacked by immune cells.
Inhibition of the “complement cascade” at the level of C3 might be a superior strategy as it prevents both hemolysis and immune cell recognition. AMY-101 inhibits complement component C3 and interrupts the complement activation cascade.
About Orphan Drug Designation
Under the EU Regulation on orphan medicinal products (Regulation (EC) No 141/2000) the EMA may grant the Orphan Drug Designation to facilitate drug development for drugs that target rare but life-threatening or chronically debilitating conditions, which potentially provide a significant therapeutic advantage over existing therapies. The first new drug application to receive EMA approval for a particular active ingredient to treat a particular disease with an EMA orphan drug designation is entitled to a ten-year exclusive marketing period in Europe for that product and for that indication.
About Amyndas Pharmaceuticals
Amyndas Pharmaceuticals S.A. and Amyndas Pharmaceuticals LLC, two transatlantic sister companies (based in Glyfada, Greece and Philadelphia, Pa., respectively)
Amyndas Pharmaceuticals SA
N. Zerva 28, Glyfada 16675, Greece
T: +30 (210) 894-1213
Dr. Despina Yancopoulou