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PHARMACY

FDA approves Catalyst’s Firdapse

BY Sandra Levy

Catalyst Pharmaceuticals has received the ok from the Food and Drug Administration for Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome, or LEMS in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS.

“There has been a long-standing need for a treatment for this rare disorder,” director of the division of neurology products in the FDA’s Center for Drug Evaluation and Research Billy Dunn said. “Patients with LEMS have significant weakness and fatigue that can often cause great difficulties with daily activities.”

In people with LEMS, the body’s own immune system attacks the neuromuscular junction, which is the connection between nerves and muscles, and disrupts the ability of nerve cells to send signals to muscle cells. LEMS may be associated with other autoimmune diseases, but more commonly occurs in patients with cancer such as small cell lung cancer, where its onset precedes or coincides with the diagnosis of cancer. The prevalence of LEMS is estimated to be three per million individuals worldwide.

The FDA granted this application Priority Review and Breakthrough Therapy designations. Firdapse also received the Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

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PHARMACY

Astellas gets FDA green light for Xospata

BY Sandra Levy

Astellas Pharma has received the Food and Drug Administration’s nod for Xospata (gilteritinib) for the treatment of adult patients who have relapsed or refractory, or resistant to treatment Acute Myeloid Leukemia, or AML with an FLT3 mutation as detected by an FDA-approved test.

Xospata is an oral therapy and the first and only FLT3-targeting therapy to be approved by the FDA for this population.

The American Cancer Society estimates that in 2018, approximately 19,000 people will be diagnosed with AML in the U.S. AML has been associated with various genetic mutations.

“Xospata offers new hope to patients for whom the treatment path forward is unclear,” Astellas senior vice president and global therapeutic area head, oncology development, Steven Benner said. “For the first time, people with relapsed or refractory FLT3 mutation-positive AML have an FDA approved FLT3-targeting treatment available to them. The approval of Xospata is also a proud, landmark moment for our oncology program and marks the first approval of a medicine that will be the cornerstone of our new presence in blood cancers.”

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FDA partners with WHO to expedite review of HIV drug applications

BY Sandra Levy

The Food and Drug Administration is working with the World Health Organization to pilot a process to share documents on HIV drug applications that have been approved or tentatively approved by the agency under the U.S. President’s Emergency Plan for AIDS Relief, or PEPFAR.

In this initial pilot, to be called the Collaborative Registration Procedure-Lite, or CRP-Lite, the FDA will, with the applicants’ permission, provide the WHO/PQP with minimally-redacted reviews of one or two HIV drug applications. The WHO/PQP will then use the FDA’s reviews to expedite its own regulatory decision making, producing review dossiers which can in turn be shared with regulators in resource limited countries to speed up their own regulatory review processes—making lifesaving drugs available to patients who need them the most.

“Since PEPFAR)was launched 15 years ago to battle the global HIV/AIDS epidemic, more than 17 million lives have been saved and currently over 14 million people living with HIV in the countries served by the program are being provided with safe, effective and low-cost antiretroviral therapy, including over 700,000 children,” FDA’s deputy commissioner for policy, planning, legislation and analysis Anna Abram said.

Abram continued,“As we mark this year’s World AIDS Day on December 1, we reflect on the progress we have made in the global fight against HIV/AIDS as well as the men, women and children living with HIV who do not have access to prevention, care and treatment. As part of our public health mission, the FDA has played an important role in supporting PEPFAR and the agency remains committed to doing what we can to facilitate timely access to these essential medicines.”

PEPFAR was launched in 2003 to address the global HIV/AIDS crisis by using U.S. funds to purchase, at low cost, antiretroviral therapies, including new combinations and formulations of medicines, for treatment in countries with limited resources that were hard-hit by the epidemic.

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