Multiple sclerosis drug gilenya (fingolimod) approved by fda

Multiple sclerosis drug gilenya (fingolimod) approved by fda

Gilenya (fingolimod) 0.5 mg daily for the treatment of relapsing forms of multiple sclerosis has been approved by the Food and Drug Administration. Swiss pharmaceutical company, Novartis, says that Gilenya is the first oral medication indicated for relapsing forms of multiple sclerosis (MS) in the United States - it reduces the frequency of MS flare-up, as well as helping slow down the build-up of some of the physical problems associated with MS.

Clinical trials involving 2,600 patients demonstrated a well-studied safety and tolerability profile, Norvartis informs. Some of the trial participants were in their seventh year of Gilenya treatment.

Nicholas LaRocca, VP, Healthcare Delivery and Policy Research, the National Multiple Sclerosis Society, said:

Today is a significant and encouraging day for people with relapsing forms of MS in the US. A new treatment option that offers significant efficacy in the convenience of a capsule is a welcome alternative to frequent injections for individuals living with this chronic disease.

Fred Lublin, MD, Saunders Family Professor of Neurology, The Corinne Goldsmith Dickinson Center for Multiple Sclerosis, Mount Sinai School of Medicine, said:

Through a novel mechanism of action, Gilenya can significantly improve clinical outcomes among patients with relapsing forms of MS. Gilenya provides significant efficacy and manageable safety when used in accordance with approved labeling, making it a valuable advancement for relapsing MS patients and the physicians who treat them.

Novartis writes that the Gilenya clinical trial that was submitted to the FDA was the largest ever for a new MS medication; it included combined findings from trials that demonstrated significant efficacy in reducing MS relapses, disability progression risk, and the total number of brain lesions detected by MRI (magnetic resonance imaging).

Trevor Mundel, MD, Global Head of Development at Novartis Pharma AG., said:

We are proud to have worked successfully with the MS community toward a shared goal of bringing a novel efficacious treatment to people with relapsing forms of MS. We are actively pursuing regulatory approval in Europe and the rest of the world.

Gilenya is a sphingosine 1-phosphate receptor (S1PR) modulator, the first in this new class of medication. The immune system of MS patients damages the covering that protects CNS (central nervous system) nerve fibers - the nerves in the spinal cord and brain. Scientists believe that Gilenya works by retaining specific lymphocytes (white blood cells) in the lymph nodes, thus reducing the immune system's attack on the central nervous system. If the white blood cells can be prevented from reaching the CNS there is less inflammatory damage to the nerve cells.

When the patient stops taking Gilenya, the white blood cell retention is reversed, Novartis explained.

Scientists stress that Gilenya does not cure multiple sclerosis; it can, however, slow down the accumulation of physical problems that MS may cause.

Clinical trials showed that Gilenya 0.5 mg reduced the number of relapses by 52% at one year compared with Avonex (interferon beta-1a IM).

Gilenya also reduced disease activity as measured by the number of new and newly enlarged T2 lesions on MRI scans compared to interferon beta-1a IM (1.6 vs 2.6, respectively, P=0.002) at one year.

In a two-year study, Gilenya demonstrated a reduction relapse rate of 54% compared to a placebo, as well as a 30% reduction of disability progression.

Researchers in both studies also reported statistically significant reductions in brain lesion activity.

In December 2009, Gilenya was submitted to the EMA (European Medicines Agency) as well as to the FDA. The EMA regulatory review, as well as others in various parts of the world are still ongoing.

What is Multiple Sclerosis (MS)?

With MS the central nervous system (CNS) is attacked by the person's own immune system. That is why MS is known as an auto-immune disease. The CNS includes the brain, spinal cord and optic nerves.

Nerve fibers are surrounded by myelin, which protects them. Myelin also helps conduct electrical signals, also termed impulses - i.e. myelin facilitates a good flow of electricity along the nervous system from the brain.

The myelin of a patient with MS disappears in several areas (multiple areas). This leaves a scar (sclerosis). Multiple Sclerosis means Scar Tissue. The areas where there is either not enough or no myelin are called plaques or lesions. As the lesions get worse the nerve fiber can break or become damaged.

When a nerve fiber has less myelin the electrical impulses received from the brain do not flow smoothly to the target nerve - when there is no myelin the nerve fibers cannot conduct the electrical impulses at all. The electrical impulses are instructions from the brain to carry out actions, such as to move a muscle. MS is the disability to get your body to do what your brain wants it to.

There are 4 courses of the disease. In each case, the MS may be mild, moderate or severe:

  • Relapsing-Remitting MS

    The patient experiences clearly-defined attacks of worsening neurologic function. These relapses are followed by remission (complete recovery periods). During the remission there is no disease progression. Other terms for relapses are flare-ups, or exacerbations. About 85% of MS patients are thought to have Relapsing-Remitting MS.

  • Primary-Progressive MS

    The patient experiences slowly worsening neurologic function from the beginning. There are no distinct relapses or remissions. The rate at which the MS progresses will vary over time. The patient will experience periods of temporary minor improvements. This type of MS is thought to affect about 10% of all MS patients.

  • Secondary-Progressive MS

    Initially it looks as though the patient has relapsing-remitting MS. However, the patient develops a secondary-progressive disease course in which the MS worsens more steadily. There may, or may not be intermittent relapses, remissions, or periods of temporary minor improvements.

    50% of patients with relapsing-remitting MS used to go on to develop this form of the disease within a decade before disease-modifying drugs became available. How long the new drugs may hold off the progression remains to be seen.

  • Progressive-Relapsing MS

    The patient experiences steadily worsening symptoms right from the start. There are clear attacks of worsening neurologic function along the way. The MS continues to progress without remissions - there might be some recovery after relapses. Only 5% of MS patients have this type.

Click here to read about multiple sclerosis in more detail.

Source: Novartis, archives

Looking at Long Term Results of Fingolimod (Video Medical And Professional 2020).

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