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MARCH 2016    A Monthly Review of Articles of Interest for the Clinical Community


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<This Month's Clinical Focus: ORPHAN DRUGS>

 

Nationwide Expanded Access Program for Patients with Duchenne Muscular Dystrophy (DMD)

Patients Can Now Receive Deflazacort, an Investigational Treatment from Marathon, at No Cost Through Medical Research Centers Across U.S.

Marathon Pharmaceuticals, a leader in developing treatments for rare diseases, announced today that it is expanding patient access to deflazacort, its investigational medicine for Duchenne Muscular Dystrophy (DMD). Through Marathon's ACCESS DMD™ program, deflazacort is now being made available to qualified patients, at no cost, through a growing network of medical research centers across the country. 

FREE CC00 LIC PIXABAY newspaper-art-154444_1280 2There is no cure for DMD and no drug currently approved in the U.S. to treat this rare disease.  FDA has granted Fast Track status, Orphan Drug designation and Rare Pediatric Disease designation for deflazacort for the treatment of DMD.  Marathon expects to submit a New Drug Application for deflazacort in May 2016 and, if approved by FDA, deflazacort could be made commercially available in the U.S. in January of 2017.  Expanded access programs like ACCESS DMD™ allow patients suffering from a life threatening disease to receive an investigational drug, under FDA authorization, prior to full FDA evaluation.

"In light of recent challenges faced by the DMD research community, we believe it is particularly important to step up as a company and get deflazacort into the hands of patients who may benefit, under the safeguards of a clinical trial protocol, while the company completes and FDA reviews the New Drug Application for marketing approval," said Dr. Jordan Dubow, a board certified neurologist and Chief Medical Officer at Marathon.

Medical centers participating in ACCESS DMD™ are up and running across the country and the number is expected to increase rapidly.   DMD is a rare disease affecting mainly young boys. It causes progressive muscle degeneration, and over time an inability to walk and conduct many daily activities. It also impairs pulmonary and cardiac function, and most patients do not live into their thirties. In clinical trials, deflazacort has shown the potential to slow muscle degeneration for patients with DMD.  Patients, families and physicians can learn more about ACCESS DMD™, including a list of clinical sites participating in the program, by visiting  http://www.AccessDMD.com or calling 1-844-800-4DMD (4363).

"The Duchenne community desperately needs safe and effective treatment options and Marathon's ACCESS DMD™ program has the potential to make a real difference for our families, who do not have the luxury of time," said Valerie A. Cwik, M.D., Executive Vice President & Chief Medical and Scientific Officer of the Muscular Dystrophy Association. "Expanded access to an important potential therapy option is a significant step forward as we work together as a community to help those with DMD live longer and grow stronger."


Deflazacort is a glucocorticoid with anti-inflammatory and immunosuppressant properties. Based on published clinical studies, it appears that deflazacort may be an important new treatment option for patients with DMD.  Side effects reported to date include cushingoid appearance, hirsutism, weight gain, erythema, nasopharyngitis, irritability and cataract formation.  Deflazacort is currently not approved in the U.S. but is available outside the U.S., where it is approved for many uses, but not for DMD. Deflazacort is an investigational medication and is therefore not yet proven to be safe and effective for the treatment of patients with DMD.

Source: Displayed with permission from PRNewswire
READ FULL ARTICLE with References HERE:  RePubHub


FDA Granting the Product License of ONIVYDE™ for the Treatment of Metastatic Pancreatic Cancer

PharmaEngine, Inc. announced that TFDA (Taiwan Food and Drug Administration) approved the product license of ONIVYDE™

FREE CC00 LIC PIXABAYcancer-news-magnifier-389921_1920ONIVYDE is indicated, in combination with fluorouracil (5-FU) and  leucovorin (LV), for the treatment of patients with metastatic adenocarcinoma of the pancreas after disease progression following gemcitabine-based therapy.  ONIVYDE ™ (irinotecan liposome injection, nal-IRI), also known as MM-398 or PEP02, is a novel encapsulation of irinotecan in a liposomal formulation. The active metabolite of irinotecan is SN-38, which functions by inhibiting topoisomerase I (an essential enzyme involved in DNA transcription and replication) and promoting cell death.

There are two steps for the marketing approval of new drugs in Taiwan. The first step is to assess whether the chemistry, manufacturing and controls, preclinical and clinical data regarding the quality, safety and efficacy are sufficient to issue the regulatory approval letter; and the second step is to assess whether the product labeling and package insert are supported by the new drug application dossiers. Both steps are essential for product sales in the Taiwan market.

ONIVYDE had been granted the TFDA regulatory approval letter on October 22, 2015. The approval of the commercial packaging materials and the GMP approval letters for two US manufacturing sites have been issued to allow the TFDA granting the product license of ONIVYDE.

"We are very grateful that the TFDA accelerated the approval of the product license in such an expedited manner," said C. Grace Yeh, Ph.D., President and CEO of PharmaEngine. "In addition, we highly appreciate our license partner, Merrimack Pharmaceuticals, Inc. for their total support during the review period."

During 2011 and 2014, Merrimack sponsored the global phase 3 NAPOLI-1 study in metastatic pancreatic cancer patients. In September 2014, Merrimack licensed ONIVYDE outside of the U.S. and Taiwan to Baxalta Incorporated, formerly Baxter International's BioScience business. ONIVYDE has received regulatory approvals by the TFDA and the US FDA and is under the Marketing Authorization Application (MAA) review by the European Medicines Agency (EMA). ONIVYDE has orphan drug designation in the US, EU and elsewhere.

Source: Displayed with permission from PRNewswire
READ FULL ARTICLE HERE:  RePubHub


Image Credits: All courtesy of Pixabay Free CC00 License


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