These PowerPoint slides reference FDA approval of new drugs for periodic paralysis, ALS, Duchenne muscular dystrophy, myasthenia gravis and spinal muscular atrophy, and are intended to be added to new or existing presentations. For questions, contact Amy Madsen, MDA marketing communications manager, at amadsen@mdausa.org.

Update: Therapy Development

Six New Treatments FDA recently has approved: Eculizumab (Soliris) – Oct. 23, 2017 Edaravone (Radicava) – May 5, 2017 Deflazacort (Emflaza) – Feb. 9, 2017 Nusinersen (Spinraza) – Dec. 23, 2016 Eteplirsen (Exondys 51) – Sept. 19, 2016 Dichlorphenamide (Keveyis) – Aug. 10, 2015 Radicava is the first drug approved to treat ALS in the United States in more than 20 years. Emflaza may be a better alternative to some existing DMD treatments. Spinraza is the first treatment available to target the underlying genetic cause of spinal muscular atrophy (SMA). Exondys 51 is the first disease-modifying drug approved to treat the underlying cause of Duchenne muscular dystrophy (DMD).

Soliris (MG) Soliris is a terminal complement inhibitor that targets a part of the immune system called the complement system. In clinical trials, people given Radicava showed significantly less decline in physical function compared to those who were given a placebo. Soliris is not a cure, but it may lessen the symptoms experienced by people living with generalized MG. Soliris was tested in clinical trials in people who had previously failed immunosuppressive treatment and continued to suffer from significant unresolved disease symptoms such as difficulties seeing, walking, talking, swallowing and breathing. Participants taking Soliris had improved scores on scales designed to assess quality of life and symptom burden including double vision, ptosis (drooping of the eyelids), swallowing, speech, breathing, and use of arms and legs.

Radicava (ALS) Thought to work by relieving the effects of oxidative stress In clinical trials, people given Radicava showed significantly less decline in physical function compared to those who were given a placebo. Depending on the level of function when someone begins treatment, the impact of Radicava demonstrated in clinical trials could translate into potentially helping people preserve function longer, prolonging their ability to get out of bed or eat unassisted.

Emflaza (DMD) Corticosteroid (also known as glucocorticoid) Works as anti-inflammatory and immunosuppressant Approved to treat all forms of Duchenne muscular dystrophy, regardless of mutation Emflaza has been shown in studies to slow the loss of muscle strength and function, preserve cardiac and respiratory function, and reduce incidence of scoliosis (spine curvature) in DMD Less severe side effects than similar drugs Importantly, the unwanted side effects often experienced with corticosteroids, such as weight gain, loss of bone mass, glucose intolerance (diabetes) and behavioral issues, may be less severe with Emflaza as compared to other steroids.

Spinraza (SMA) “Antisense” drug designed to slow disease progression in spinal muscular atrophy First disease-modifying treatment for SMA to be approved by FDA Expected to help all kids and adults with SMA Administered by intrathecal injection (needle injection into the fluid surrounding the spinal cord; also called a lumbar puncture), Spinraza targets genetic instructions at the RNA stage (an intermediate step between DNA and the protein manufacturing stage inside cells), resulting in increased production of the needed survival motor neuron (SMN) protein. Spinraza has been approved by the FDA to treat all forms of chromosome 5 SMA caused by a deficiency of the SMN protein. In clinical trials, treatment with Spinraza led to clinically meaningful improvement in motor function compared to treatment with a placebo. In open-label studies, some patients achieved milestones such as the ability to sit unassisted, stand or walk when they would otherwise be unexpected to do so and maintained milestone abilities at ages when it is expected they would be lost.

Exondys 51 (DMD) “Exon skipping” drug designed to slow disease progression in some forms of DMD First disease-modifying treatment for DMD to be granted accelerated approval by the FDA Expected to help approximately 13 percent of kids and adults with Duchenne Administered by intravenous infusion, Exondys targets a section of genetic code called “exon 51” in the dystrophin gene. Individuals with DMD caused by a mutation that would be impacted by skipping exon 51 could benefit from treatment with Exondys 51. Treatment with Exondys will not cure Duchenne. It could, however, slow progression, lessening muscle weakness and extending the ability to walk, to eat independently and to breathe without assistance.

Keveyis (Periodic Paralysis) The only prescription medication for hyperkalemic, hypokalemic and related variants of primary periodic paralysis Has proven effective in helping people gain greater control over the disease by decreasing the number, severity and frequency of attacks

Impact of Approvals Even as we pause to celebrate recent successes, MDA is working hard to drive more therapies across the finish line. FDA approval of these drugs is expected to encourage drug development in the neuromuscular disease space. At MDA, we’re working hard every day to find solutions, and we won’t stop until treatments are available for all the diseases in our program.

MDA Support Leads to Success MDA has supported research and clinical study into the effects of cortico-steroids including Emflaza. MDA support to Adrian Krainer at Cold Spring Harbor Laboratory led to the develop-ment of Spinraza. MDA supported development of Exondys 51 via funding to Steve Wilton at Murdoch University, Australia. MDA support helped fund early-stage development and phase 3 testing to help make Keveyis possible. As a result of our more than $1 billion dollars of research investments over the last 65 years, MDA is connected to nearly every major advance in neuromuscular disease research – including development of Emflaza, Spinraza, Exondys 51 and Keveyis.

Our sense of urgency has never been greater as we continue, with deepest thanks to all of those who have supported us along the way, to fight for strength, independence and life.