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Genzyme announced on Monday that the Food and Drug Administration has granted Fast Track designation for the development of GZ/SAR402671, a new investigational therapy for the treatment of Fabry disease.

FDA’s Fast Track Drug Development Program is designed to facilitate frequent interactions with the FDA review team to expedite the clinical development and review of a New Drug Application (NDA) for medicines with the potential to treat serious or life-threatening conditions and address unmet medical needs for such disease or conditions. It also provides the opportunity to submit sections of an NDA on a rolling basis before a sponsor submits the complete application. Genzyme is currently enrolling patients in its Phase 2a trial of GZ/SAR402671, and plans to enroll nine treatment-naïve male adult patients with Fabry disease in this international, multicenter study.

Fabry disease is an inherited and progressive condition that is characterized by excessive accumulation of the lipid GL-3 in various organs and tissues. Early symptoms include significant pain, gastrointestinal disturbances, as well as other manifestations, and over time, patients may experience life threatening renal, cardiac and cerebrovascular events. As a result, patients with Fabry disease typically have a shortened life span. Fabry disease affects both males and females, with approximately 10,000 diagnosed patients in the world.

“Becoming a Fast Track Program is an important milestone and we appreciate this designation from the FDA,” said Genzyme’s acting Head of Rare Diseases, Richard Peters, MD, PhD. “We look forward to learning more about this small molecule, with the goal of providing more therapeutic options to the Fabry community as quickly as possible.”

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Nearly 5,000 transplant professionals will gather in Philadelphia later this week for the 15th annual American Transplant Congress (ATC). The event is the joint meeting of the American Society of Transplant Surgeons and the American Society of Transplantation, both non-profit medical associations for professionals involved in scientific research and advocacy related to the advancement of transplantation.

The multitude of programs featured during the ATC meeting are designed for physicians, surgeons, scientists, nurses, organ procurement personnel, advanced transplant providers, pharmacists, administrators and allied health professionals interested in solid organ and tissue transplantation. Throughout the five-day event, the scientific and clinical knowledge of worldwide transplant professionals will be challenged and stretched as a variety of groundbreaking research is presented.

The pre-meeting symposia, being held on Saturday, May 2, will offer a variety of courses for clinicians, scientists, and allied health professionals and will focus on key advances incurrent topics of major interest in the scientific, surgical, and clinical practices of transplantation.

“The 2015 American Transplant Congress offers professionals the opportunity to exchange new scientific and clinical information relevant to solid organ and tissue transplantation with others in the field,” said Rob Fairchild, PhD, Congress co-chair. “The meeting gives attendees an opportunity like no other—to network and collaborate with peers and identify future opportunities that can lead to major advancements in transplantation.”

The program will feature:

• Specific tracks designed to cater to professionals focused on basic science, pediatric transplantation, immunology, tissue injury/preservation, xenotransplantation, pathology, nephrology, organ donation, infectious diseases, heart transplantation, immunosuppression, liver transplantation, and allied health.
• More than 1,700 abstracts, nearly 530 concurrent oral sessions, and close to 2,000 poster presentations.

“We are very excited about the innovative, cutting-edge science that will be presented at this year's ATC,” said Dorry Segev, MD, PhD, Congress co-chair. “Novel findings will be presented and highly controversial subjects will be addressed in topics ranging from multi-organ transplantation, generic immunosuppression, liver allocation and policy, living donation, kidney exchanges/chains, and transplantation in the elderly, to artificial organs, HIV and hepatitis C, technical challenges, new biomarkers, and tolerance.”

Additional program information can be found at http://2015.atcmeeting.org/.

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KOZHIKODE: Dr Ebin Soorya, one of the Keralite doctors injured in the Nepal quake, was subjected for a dialysis. Ebin underwent dialysis after doctors said his renal condition was not good. He will be airlifted to Delhi for better treatment. The relatives of Irshad will leave for Nepal Tuesday afternoon.

Ebin will be brought to New Delhi by noon, representatives said.  12 more Keralites have reached Delhi from Nepal. So far 250 Keralites have reached, authorities said.

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