Novo Nordisk drug for rare hemophilia gets FDA nod
SILVER SPRING, Md. — The Food and Drug Administration has approved a new treatment made by Danish drug maker Novo Nordiskfor a rare, genetic bleeding disorder, the agency said Monday.
The FDA announced the approval of Tretten (coagulation factor XIII A-subunit [recombinant]), calling it the first recombinant product for routine prevention of bleeding in adults and children with congenital factor XIII A-subunit deficiency. Factor XIII is a protein involved in blood clotting, and patients with the condition don’t make enough of it. The protein comprises two subunits, A and B; most cases of factor XIII deficiency result from a deficiency of the A-subunit. According to the National Hemophilia Foundation, factor XIII deficiency may be the rarest of all factor deficiencies, affecting one-in-5 million people.
"The approval of this product provides another therapeutic option for the prevention of bleeding in patients with factor XIII A-subunit deficiency," FDA Center for Biologics Evaluation and Research director Karen Midthun said. "Without treatment, people with this rare condition are at risk for serious and life-threatening bleeding."
FDA move allows Ariad Pharmaceuticals’ resumed marketing of Iclusig
CAMBRIDGE, Mass. — The Food and Drug Administration has approved revisions to the labeling of a leukemia drug made by Ariad Pharmaceuticals that allow the company to resume selling it, the company said.
Ariad said the FDA approved revised prescribing information and a risk evaluation and mitigation strategy, or REMS, for Iclusig (ponatinib) in refractory Philadelphia chromosome-positive, or Ph+ leukemias. These conditions include T315I-positive chronic myeloid leukemia; T315I-positive Ph+ acute lymphoblastic leukemia; and chronic-phase, accelerated-phase or blast-phase chronic myeloid leukemia; or Ph+ acute lymphoblastic leukemia in patients for whom no other similar therapy can be used.
"In less than two months of suspending marketing and commercial distribution of Iclusig in the [United States], we addressed the issues raised by the FDA and now are able to market and distribute Iclusig again in the [United States]," Ariad chairman and CEO Harvey Berger said. "As we look ahead to relaunching Iclusig in the [United States] and fulfilling our post-marketing requirements, we will continue to focus on understanding the benefits and risks of Iclusig treatment in patients with resistant or intolerant Philadelphia-positive leukemias."
CMS outlines recommendations to smooth Obamacare transition
ARLINGTON, Va. — The Centers for Medicare and Medicaid Services is encouraging health plans to cover patients’ existing non-formulary drugs as though they were formulary drugs for the first 30 days after the Jan. 1 rollout of the insurance coverage provided under the Patient Protection and Affordable Care Act.
CMS also is recommending that plans treat out-of-network providers as in-network providers during the initial months of enrollment to address situations in which out-of-network providers were listed as in-network at the time of enrollment or are providing treatments for acute episodes of care at the start of the year.
The recommendations have drawn support from the National Association of Chain Drug Stores, a trade group of retail pharmacy chains, which is hoping that CMS includes pharmacies as part of the recommendations.
"We welcome the additional millions of Americans who will have new insurance coverage," a letter from the NACDS to the CMS read. "We are committed to working with all parties to address challenges and to maximize the experience of patients, similar to the efforts of community pharmacy that were noted as vital to smoothing the transition during the rollout of Medicare Part D."
The group also is recommending that CMS "empower patients with information" so that they can maximize their experience with healthcare reform.
"We are concerned that the initial enrollment deadline extensions may result in some newly insured patients not having the information necessary to process pharmacy claims for them in January," the NACDS’ letter read. "We are also concerned that some patients who enroll in a plan and take the opportunity to pay January premiums later in January may not be aware that they must pay for their health care and prescription drug services out-of-pocket pending payment of their insurance premium. In light of our concerns, NACDS urges CMS and insurers to engage in consumer education and outreach to address and mitigate these scenarios, offer ample support for pharmacies via call centers throughout the holidays and use techniques that helped to ease the transition for the initial enrollment in Medicare Part D."