FDA Committee Recommends the Approval of FTY720
The oral medication FTY720 (fingolimod) has moved another step closer to approval as a new disease-modifying therapy for MS. The United States Food and Drug Administration (FDA) Peripheral and Central Nervous System Drugs Advisory Committee reviewed the clinical trial data submitted by Novartis Pharmaceuticals Corporation (makers of FTY720), and reported its findings at a meeting held on June 10, 2010.
At a count of 25 to 0, the vote was unanimous in favor of recommending FTY720 for approval. This was based on the effectiveness and safety data of the proposed 0.5 mg dose (the lower of the two dose levels studied) for individuals with relapsing forms of MS. This includes the relapsing-remitting form of MS, as well as secondary-progressive MS with relapses.
While a recommendation by the FDA’s advisory committee does not guarantee approval, it does play an important role with the FDA’s final decision, which is presently scheduled to be announced in September 2010. Initially, FTY720 had been granted priority review status by the FDA, reducing the FDA’s review process from 10 months down to six months. A decision was tentatively scheduled for June 2010, however, a three-month extension was given to allow more time for review. This extension was anticipated by Novartis, since FTY720 contains an ingredient which has not been approved before (a "New Molecular Entity").
If the drug is approved, the FDA’s advisory committee recommends that Novartis continues to monitor safety by collecting data on patients taking FTY720. Drug-related adverse events include: a reduction in heart rate (dose-related and transient); infrequent transient AV conduction block of the heart; a mild increase in blood pressure; macular edema (an eye condition that can affect vision, more common with the higher dose); reversible elevation of liver enzymes; and a slight increase in lung infections (primarily bronchitis). Novartis plans to work closely with the FDA as they determine what types of evaluation and intervention will be necessary before and during drug treatment to address these potential adverse events.
The FDA’s advisory committee also recommends that a post-marketing evaluation of a lower dose of FTY720 be conducted. This would determine if a lower dose could offer similar effectiveness, while providing greater safety from the above-mentioned potential adverse events.
Seven disease-modifying therapies have been FDA approved and are presently available by prescription. These are given via self injections at the patient’s home, or by infusion at a medical facility. If approved, FTY720 would be the first oral medication available for the long-term treatment of MS. (One other oral medication for MS, oral cladribine, has recently been resubmitted with a New Drug Application to the FDA. Several other oral medications are also in development.) In the clinical trials, FTY720 was in capsule form, given once daily by mouth (orally). A brand name for the drug has not yet been selected.
FTY720 presents a new option for individuals with MS. While many members of the MS community presently take one of the approved disease-modifying therapies and are doing very well on the therapy, not everyone responds to or is able to tolerate these medications. Particularly for these individuals, and for people who are uncomfortable with injections and/or experience injection-site reactions, the prospect of a new oral medication for MS is very encouraging.
At the FDA advisory committee meeting on June 10th, members of the MS community spoke about the importance of a new treatment option. Representatives of MS organizations attended as well, including MSAA President and CEO Doug Franklin, who noted MSAA’s support for the development of new investigational drugs aimed at treating MS. He also stressed the importance of treatment safety and ensuring that proper steps are taken to minimize any risks.
FTY720 has been shown to reduce relapse rate and delay the progression of disability, as well as reduce brain lesion activity and brain volume loss, as measured by magnetic resonance imaging (MRI), when compared to control groups given a placebo. A one-year study also compared its effectiveness to interferon beta-1a (Avonex®). For specific information on these trials, please refer to MSAA’s website article, "Positive Study Results with Oral FTY720 Leads to Granting of Priority Review Status by FDA."
For more information, please call MSAA’s Helpline at (800) 532-7667.
Written by Susan Wells Courtney, MSAA Senior Writer
Reviewed by Jack Burks, MD, MSAA Chief Medical Officer