Healthcare Industry News: orphan drug designation
News Release - June 21, 2016
Savara Pharmaceuticals Acquires Serendex PharmaceuticalsAdds two orphan respiratory drugs to its growing pipeline
AUSTIN, Texas, June 21, 2016 -- (Healthcare Sales & Marketing Network) -- Savara Pharmaceuticals, an emerging specialty pharmaceutical company focused on rare respiratory diseases, today announced that the company has acquired the assets of Serendex Pharmaceuticals adding two new programs to its pipeline. The lead product of Serendex, Molgradex®, is an inhaled form of granulocyte-macrophage colony-stimulating factor (GM-CSF), currently in a Phase II/III clinical study in Europe and Japan to treat autoimmune pulmonary alveolar proteinosis (PAP). The acquisition is subject to customary closing conditions, including approval by Savara's shareholders.
"The acquisition is a transformational milestone for Savara, expanding our pipeline of orphan respiratory drugs with an exciting product for a highly debilitating disease with no approved medicinal treatments," said Rob Neville, Chief Executive Officer of Savara Pharmaceuticals. "Molgradex® is a beautiful fit with our lead product, AeroVanc, and has the potential to be a truly disease-modifying treatment for PAP based on clinical evidence from academically sponsored pilot studies and case reports on inhaled GM-CSF."
Molgradex® is a nebulized form of recombinant human GM-CSF, delivered using the PARI eFlow high-efficiency rapid nebulizer system. The lead indication of Molgradex®, autoimmune PAP, is caused by autoantibodies that disrupt a key function of GM-CSF in the lungs, recycling of the lung surfactant. The functional GM-CSF deficiency causes excessive surfactant accumulation in the lungs, leading to decreased gas exchange, shortness of breath and even respiratory failure. PAP affects approximately 10,000 people in the main global pharmaceutical markets. Serendex has recently begun enrollment in a Phase II/III trial of Molgradex®, which will allow submission of a marketing authorization application in Europe and Japan.
In addition to PAP, Molgradex® has potential in other indications by stimulating the innate immune system to enhance microbial killing or repair of impaired lung tissue. The second product acquired in the deal is an inhaled form of Factor VIIa, a promising preclinical-stage product for a rare lung disease called diffuse alveolar hemorrhage (DAH). DAH is a potentially life threatening form of bleeding into the alveoli of the lungs where oxygen exchange takes place.
About Savara Pharmaceuticals
Savara Pharmaceuticals is a specialty pharmaceutical company focusing on innovative drugs for the treatment of serious and life-threatening rare respiratory diseases.
The company's lead product, AeroVanc, is the first dry powder inhaled antibiotic being developed for the treatment of persistent MRSA infection in people with Cystic Fibrosis (CF). By delivering vancomycin directly to the lungs, higher vancomycin concentrations are achieved at the site of infection, which is expected to lead to improved clinical efficacy. In addition, direct delivery of the drug into the lungs reduces exposure to the drug elsewhere in the body, and is thereby expected to reduce the risk of systemic drug-related side effects. Infection by MRSA has become increasingly common with a prevalence of almost 30 percent of the estimated 32,000 people with CF in the U.S. Persistent MRSA infection is associated with faster decline in lung function, increased hospitalizations and reduced survival. Currently there is no approved inhaled therapy for MRSA infection in people with CF.
AeroVanc has received from the FDA Fast Track and orphan drug designations as well as Qualified Infectious Disease Product (QIDP) status providing a total of 12 years of market exclusivity. Enrollment for the pivotal Phase III study of AeroVanc is expected to begin in late 2016.
Visit www.savarapharma.com or www.aerovanc.com to learn more or find us on Twitter, Facebook and LinkedIn.
About Serendex Pharmaceuticals
Established in 2008 in Copenhagen, Denmark, Serendex Pharmaceuticals has focused on developing novel inhalation therapies for the treatment of patients with rare respiratory conditions. In 2014 Serendex listed on the Oslo Stock Exchange, and operated as a public company until a recent delisting on May 4th, 2016.
After a successful Phase I clinical trial of GM-CSF for PAP, Serendex received positive EMA scientific advice for an accelerated clinical pathway, allowing the company to submit a marketing approval application based on a single placebo-controlled study. This pivotal Phase II/III clinical study (the IMPALA study) begun enrollment in May 2016 in Europe and Japan.
Serendex have recently entered into a milestone and royalty bearing license agreement with Japanese Nobelpharma Co., Ltd for future commercialization of Molgradex® in Japan. Since 2015, Molgradex® is available for use in selected PAP patients in Denmark, Germany and Austria under the Early Patient Access program.
For more information, visit www.serendex.com or pap.info.
Source: Savara Pharmaceuticals
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