Granted for Three Distinct Indications: Chronic Pulmonary Infections Due to Pseudomonas Aeruginosa in Patients with Cystic Fibrosis and in Patients with Non-Cystic Fibrosis Bronchiectasis as well as in Patients with Nontuberculous Mycobacteria
Pre-NDA Discussion with FDA in Cystic Fibrosis Scheduled for 2Q 2016
NOVATO, Calif., March 16, 2016 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced that the U.S. Food and Drug Administration (FDA) has granted Qualified Infectious Disease Product (QIDP) designation to MP-376, the company's proprietary inhaled levofloxacin, for three distinct indications: for the treatment of chronic pulmonary infections due to Pseudomonas aeruginosa, in patients with cystic fibrosis (CF) and in patients with non-cystic fibrosis bronchiectasis (BE), and in patients with nontuberculous mycobacteria (NTM).
Created under the Generating Antibiotics Incentives Now (GAIN) Act of 2012, the QIDP designation was established for the purpose of encouraging pharmaceutical companies to develop new antimicrobial drugs to treat serious and life-threatening infections. Granting of the QIDP designation by FDA highlights the potential of MP-376 to address a number of serious and life-threatening infections and provides Raptor with significant incentives for the development of MP-376, including priority review by the FDA, eligibility for Fast Track designation and a five-year extension of marketing exclusivity under the Hatch-Waxman Act. QIDP also paves the way for FDA to apply greater regulatory flexibility in the case of high unmet medical need for serious life-threatening infections. Raptor holds orphan designation in the U.S. for MP-376 for the treatment of CF, which, if granted after approval, confers seven years market exclusivity.
"We are pleased with the FDA's decision to grant QIDP designation to MP-376 as we believe this drug has the potential to provide a significant benefit in the treatment of P. aeruginosa infections in patients with CF and in other chronic pulmonary infections in patients with bronchiectasis and nontuberculous mycobacteria," said Krishna Polu, M.D., Chief Medical Officer of Raptor Pharmaceutical Corp. "Importantly, this designation validates the unmet need caused by a growing number of persistent lung infections which can result in death in these patients. QIDP designation will help to facilitate the development of MP-376 in these indications. We look forward to having regulatory discussions with the FDA in CF in the second quarter and initiating a Phase 2 program in bronchiectasis this year."
About MP-376 (inhaled levofloxacin hemihydrate)
MP-376 is the investigational form of QUINSAIRTM, a proprietary inhaled formulation of levofloxacin, a fluoroquinolone antibiotic, which is being developed for the management of chronic pulmonary infections due to P aeruginosa in patients with CF in the U.S. QUINSAIR is approved in the EU and in Canada for the management of chronic pulmonary infections due to P. aeruginosa in adult patients with CF. MP-376 is also being developed for the treatment of non-CF bronchiectasis and NTM. Raptor plans to initiate a Phase 2 study in non-CF bronchiectasis by the end of 2016. Work to support further clinical development in lung infections associated with NTM is also planned. Administration of MP-376 with a high efficiency eFlow Nebulizer System (PARI Pharma GmbH) allows for the delivery of high concentrations of active drug directly to the site of infection in approximately five minutes.
About Raptor Pharmaceutical
Raptor Pharmaceutical Corp. is a global biopharmaceutical company focused on the development and commercialization of transformative therapeutics for rare, debilitating and often fatal diseases. With its recent acquisition of QUINSAIR (MP-376), Raptor plans to to seek approval of MP-376 for CF in the U.S., and to pursue clinical programs in both non-CF bronchiectasis and NTM. In addition, Raptor is the market leader in the commercialization of RP103 (known commercially as PROCYSBI) in nephropathic cystinosis in the U.S. and Europe. Raptor is developing RP103 in additional multiple therapeutic areas such as Huntington's disease and mitochondrial disorders including Leigh syndrome. Raptor holds several orphan drug designations, including orphan drug exclusivity for nephropathic cystinosis in the U.S. and EU. For additional information, please visit www.raptorpharma.com.
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements are indicated by words or phrases such as "believes," "expects," "anticipates," "estimates," "plans," "continuing," "ongoing," "projected" and similar words or phrases and relate to future events, including statements regarding: PROCYSBI as a treatment option for patients with nephropathic cystinosis, Huntington's disease and mitochondrial disorders, including Leigh syndrome, orphan drug exclusivity for the therapy in the U.S., Raptor's plans to develop MP-376 in cystic fibrosis, bronchiectasis not associated with cystic fibrosis and nontuberculous mycobacteria,the timing and outcome of those development programs and any discussions with regulatory authorities and Raptor's other development programs. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause the company's actual results to be materially different from these forward-looking statements. Raptor cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Factors which may contribute to differences in actual results include, among others: Raptor's ability to market and sell QUINSAIR; continued and increased market acceptance and sales of PROCYSBI in the U.S. and other territories; Raptor's ability to expand the use of RP103 and MP-376 and to receive regulatory approval for other indications; Raptor's reliance on single active pharmaceutical ingredient suppliers for PROCYSBI and QUINSAIR and other third parties in connection with drug product development; compliance with healthcare regulations, ongoing regulatory requirements and potential penalties; any serious adverse side effects associated with PROCYSBI, QUINSAIR or any other future products; any product liability claims; third-party payor coverage, reimbursement and pricing for PROCYSBI, QUINSAIR and future products; enacted and future healthcare legislation; Raptor's ability to obtain and maintain orphan drug or other regulatory exclusivity for PROCYSBI, QUINSAIR or any other future products; the integration of European operations with U.S. operations; relationships with key scientific and medical collaborators; intellectual property protection and claims and continued license rights; and Raptor's ability to fund its operations and make required payments on its debt. Certain of these risks, uncertainties and other factors are described in greater detail in the company's filings from time to time with the Securities and Exchange Commission (SEC), which Raptor strongly urges you to read and consider, including: Raptor's annual report on Form 10-K for the twelve months ended December 31, 2015 filed with the SEC on February 26, 2016, Raptor's quarterly reports on Form 10-Q for the quarterly periods ended March 31, 2015, June 30, 2015 and September 30, 2015 filed with the SEC on May 7, 2015, August 6, 2015 and November 5, 2015, respectively, and Raptor's other periodic reports filed with SEC, all of which are available free of charge on the SEC's web site at http://www.sec.gov. Subsequent written and oral forward-looking statements attributable to Raptor or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in Raptor's reports filed with the SEC. Raptor expressly disclaims any intent or obligation to update any forward-looking statements except as may be required by law.
Kimberly Lee, D.O.
Vice President, Corporate Strategy and Communications
Raptor Pharmaceutical Corp.
Westwicke Partners, LLC
Robert H. Uhl