• Alexion Pharmaceuticals Inc., of Cheshire, Conn. has received approval from the FDA to produce Soliris (eculizumab), a treatment for paroxysmal nocturnal hemoglobinuria, at its Rhode Island manufacturing facility. Alexion will continue to supply Soliris from its contract manufacturer, while ramping up additional production through the Rhode Island facility. The company has already received approval from the European Medicines Agency to supply Soliris to European markets, and is seeking similar approvals in other countries.

• Alnylam Pharmaceuticals Inc., of Cambridge, Mass., and collaborators at the Parkinson's Institute and the Mayo Clinic have published new research findings in the journal Public Library of Science in therapeutic silencing of Parkinson's disease. The new data showed effective silencing of the alpha-synuclein gene with an RNAi therapeutic administered directly to the substantia nigra in the CNS of non-human primates. Alpha-synuclein is widely believed to play a central role in the development of Parkinson's disease. To date, no drugs have been identified that are capable of lowering alpha-synuclein levels. The study was conducted in collaboration with scientists at Mayo Clinic and the Parkinson's Institute, and funded by a LEAPS (Linked Efforts to Accelerate Parkinson's Solutions) award from The Michael J. Fox Foundation for Parkinson's Research. Alnylam has an agreement with the Mayo Clinic whereby Mayo has granted Alnylam an exclusive license to certain patents and know-how related to alpha-synuclein.

• AVI BioPharma Inc., of Bothell, Wash., said that preclinical data published online in Nature Medicine demonstrated postexposure efficacy of AVI's candidates against Ebola and Marburg viruses. AVI-6002 and AVI-6003, the company's respective lead therapeutic candidates against Ebola and Marburg viruses, both employing AVI's advanced PMOplus chemistry, provided postexposure efficacy in non-human primates. Treatment of Ebola virus-infected primates with AVI-6002 led to 60 percent survival, and treatment of Marburg-infected primates with AVI-6003 conferred 100 percent survival, compared to control groups where both viruses were universally lethal. The work was a collaborative effort between AVI BioPharma and scientists at the U.S. Army Medical Research Institute of Infectious Diseases, the Department of Defense's lead medical research laboratory for biological defense.

• BIND Biosciences Inc., presented its Medicinal Nanoengineering platform at the American Chemical Society National Meeting, including results from preclinical studies on its lead nanomedicine drug, BIND-014, which is set to enter clinical trials later this year. BIND-014 is a targeted, polymeric nanoparticle containing the cytotoxic agent, docetaxel (Taxotere, Sanofi Aventis SA), which is approved in major cancer indications, including breast, prostate and lung. In animal models of human cancers, BIND-014 was shown to deliver up to 20 times more docetaxel to the tumor site than an equivalent dose of Taxotere.

• Cumberland Pharmaceuticals Inc., of Nashville, Tenn., said the FDA extended its review of the firm's supplemental new drug application for Acetadote (acetylcysteine) in non-acetaminophen acute liver failure. The PDUFA date now is set in December.

• MannKind Corp., of Valencia, Calif., will provide its ultra rapid-acting insulin, Afrezza, for use in a closed-loop blood sugar monitoring and insulin delivery system being studied by the Juvenile Diabetes Research Foundation as part of its Artificial Pancreas Project. The Sansum Diabetes Research Institute and the University of California, Santa Barbara are additional partners in the study. Affreza was chosen for the study because its pharmacokinetic profile is closest to the human body's natural insulin.

• Pfizer Inc., of New York, reported that the World Health Organization granted prequalification to Prevenar 13 (pneumococcal polysaccharide conjugate vaccine, 13-valent, adsorbed) for active immunization of infants and children ages 6 weeks through 5 years against invasive disease, pneumonia and otitis media caused by the 13 pneumococcal serotypes contained in the vaccine. WHO prequalification allows for the procurement of Prevenar 13 by United Nations agencies, including the United Nations Children's Fund, governments and other organizations for use in national immunization programs. The prequalification is for global use of the vaccine in a single-dose vial.

• Soligenix Inc., of Princeton, N.J., was awarded a three-year, $1.2 million clinical research grant by the FDA's Office of Orphan Products Development, which the firm said will defray a significant portion of costs associated with its ongoing confirmatory Phase III trial of orBec in acute gastrointestinal graft-vs.-host disease. The SUPPORTS (Sparing Unnecessary Prednisone Phase 3 orBec Randomized Treatment Study) trial is enrolling about 166 patients, with the primary endpoint of treatment failure rate at day 80. The study is expected to complete in the first half of 2011 .

• To-BBB Technologies BV, of Leiden, the Netherlands, said the FDA granted orphan drug designation for its lead brain cancer product, 2B3-101 . That status provides regulatory assistance and would guarantee seven years of marketing exclusivity upon approval. 2B3-101 , which is ready to start Phase I/II testing, is designed to use glutathione to enhance the delivery of doxorubicin across the blood-brain barrier.