Mithridion’s PSP treatment granted orphan drug designation
MADISON, Wis. — The Food and Drug Administration has granted orphan drug designation to a treatment made by Mithridion for a rare neurological disorder, Mithridion said Monday.
The FDA gave the designation to MCD-386CR, an investigational drug for progressive supranuclear palsy, or PSP, which recently completed early-stage clinical trials. PSP is a progressive brain disease in which neurons in the regions of the brain that control eye movements, balance, walking, speech and cognition degenerate, leading to slowed thinking and difficulties with reasoning, planning and shifting between tasks.
“Orphan status will help us immeasurably to bring together the resources and support needed to evaluate MCD-386CR in this rare but important disease and indeed other devastating brain diseases for which there are no current therapies,” Mithridion CEO Trevor Twose said. “Based on results in preclinical studies, we believe MCD0386CR potentially will help restore cognition, so vital to human functioning, and potentially could treat the underlying processes causing the degeneration of neurons in PSP.”
The FDA gives orphan drug designation to investigational treatments for diseases that affect fewer than 200,000 people in the United States.
Managed Health Care Associates supports NCPA’s LTC division
ALEXANDRIA, Va. — The National Community Pharmacists Association’s long-term care division will receive significant financial support from the Managed Health Care Associates, the NCPA announced Monday.
MHA is one of the largest group purchasing organizations in the country focused on alternate site/non-acute settings with more than $5 billion in total purchasing volume to these providers, NCPA said. MHA also is an advocate for “closed door” pharmacies, which exclusively serve LTC facilities and their residents and are represented by NCPA’s LTC division, which was established in October 2010.
“NCPA has achieved notable success in its advocacy efforts on behalf of community pharmacies,” MHA CEO Douglas Present said. “We are very pleased with NCPA’s stated goal of replicating those successes for independent LTC pharmacies. We are supporting NCPA’s new LTC division because of the value and potential it offers for our membership and for all LTC pharmacies.”
Added NCPA president Robert Greenwood, “We greatly appreciate MHA’s vote of confidence in this new effort. Working together, I know our organizations will help achieve many more victories for long-term care patients and pharmacies in the months and years ahead.”
FDA seeks input from stakeholders, public on regulatory approval pathway for follow-on biologics
SILVER SPRING, Md. — The Food and Drug Administration is moving forward in the creation of a regulatory approval pathway for follow-on biologics and is requesting input from stakeholders and the public, the agency said Monday.
The FDA plans to use the input it gets to create a user fee program for regulatory approval applications.
In addition to accepting written comments, the agency will consult with scientific and academic experts, healthcare professionals, patient and consumer advocacy groups and companies.
A Federal Register notice displayed Monday described the principles the agency proposes to develop the program, a proposed structure for it and proposed performance goals. The notice is available here.