Additional incentives provided by orphan drug designation include protocol assistance, a type of scientific advice specific for designated orphan medicines, and ten years of market exclusivity once regulatory approval is received for the target indication. Fee reductions are also available depending on the status of the company and the type of service required. The orphan drug designation does not impact the regulatory requirements for obtaining approval and does not guarantee that marketing approval will be received.Soliris® is a registered trademark of Alexion Pharmaceuticals, Inc. About RA101495 Ra Pharma is developing RA101495 for paroxysmal nocturnal hemoglobinuria (PNH), refractory generalized myasthenia gravis (rMG), and lupus nephritis (LN). The product is designed for convenient, subcutaneous self-administration. RA101495 is a synthetic, macrocyclic peptide discovered by Ra's Proprietary Extreme Diversity™ platform. The peptide binds complement C5 with subnanomolar affinity and allosterically inhibits its cleavage into C5a and C5b upon activation of the classical, alternative or lectin pathways. RA101495 also directly binds to C5b, disrupting the interaction between C5b and C6 and preventing assembly of the membrane attack complex (MAC). This activity defines a novel mechanism for the inhibition of C5 function. Repeat dosing in vivo has demonstrated sustained and predictable inhibition of complement activity with an excellent safety profile. About the Extreme Diversity™ Platform Ra Pharma's proprietary Extreme Diversity™ platform allows us to produce synthetic macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small molecules. We utilize a process called ''mRNA display'' to produce extremely large and diverse libraries of peptides from which to screen for potential product candidates. The platform generates highly specific and stable peptide-like molecules with the potential for greatly increased bioavailability, improved cell permeability, and the opportunity to address protein-protein interactions including previously undruggable targets. It can produce libraries of 10 to 100 trillion members, allowing for the rapid discovery of highly potent candidate molecules.
About Ra PharmaceuticalsRa Pharmaceuticals is a clinical stage biopharmaceutical company focusing on the development of next-generation therapeutics for diseases of complement dysregulation and for a variety of orphan indications. The Company discovers and develops peptides and small molecules to target key components of the complement cascade. For more information, please visit: www.rapharma.com. Forward-Looking Statements This press release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding the safety, efficacy and regulatory and clinical progress of our product candidates, including RA101495. All such forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include the risks that Ra Pharma's product candidates, including RA101495, will not successfully be developed or commercialized; as well as the other factors discussed in the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections in Ra Pharma's Registration Statement on Form S-1 (File No. 333-214242), as amended, which is on file with the Securities and Exchange Commission and was declared effective on October 25, 2016, as well as other risks detailed in Ra Pharma's subsequent filings with the Securities and Exchange Commission. There can be no assurance that the actual results or developments anticipated by Ra Pharma will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Ra Pharma. All information in this press release is as of the date of the release, and Ra Pharma undertakes no duty to update this information unless required by law.