New drugs approved in 2017 for serious central nervous system diseases

(January 14, 2018)

While no cures have yet been found for the following four devastating neurological diseases, in 2017 each of them received an approval for a new treatment option. Costs, as expected, are very high, as are risks of side effects. The diseases (Parkinson's, Multiple Sclerosis, Huntington's, and ALS) are listed in descending order of prevalence in the U.S.

Parkinson's Disease

Patient population: 500,000 in the U.S. with 50,000 new diagnoses per year
Notable Patients: Michael J.Fox (actor), Billy Graham (minister), Muhammad Ali (boxer)
New Drug: Xadago (safinamide)
Company: US WorldMeds LLC
Estimated Cost: $600-$800 per month
Administered: Pill taken once daily FDA press release states this is an "add-on treatment" for patients with Parkinson's disease who are currently taking levodopa/carbidopa and experiencing "off" episodes. An "off" episode is a time when a patient's medications are not working well, causing an increase in Parkinson's symptoms, such as tremors and difficulty walking.
Disease info: NIH Neurological Institute Parkinson's Disease Information Page

Multiple Sclerosis

Patient population: 250,000 - 350,000 in the U.S. with 10,000 new diagnoses per year
Notable Patients: Montel Williams (talk show host), Terri Garr (actress)
New Drug: Ocrevus (ocrelizumab)
Company: Genentech
Estimated Cost: $65,000 per treatment
Administered: Intravenous infusion; initial dosing is taken 2 weeks apart, and subsequent doses are given every 6 months. Treats patients with relapsing-remitting (most common) and primary progressive forms of multiple sclerosis.
Disease info: NIH Neurological Institute Multiple Sclerosis Information Page

Huntington's Disease

Patient population: 30,000 in the U.S. with 150,000 more having a 50% chance of being diagnosed
Notable Patients: Woodie Guthrie (folk singer)
New Drug: Austedo (deutetrabenazine)
Company: Teva Pharmaceuticals
Estimated Cost: $60,000 per year
Administered: Pill taken with food once a day and later in the treatment twice a day. This drug treats chorea (uncontrolled movements), which get worse as the disease progresses. According to the HDSA web site, many describe the symptoms of HD as having ALS, Parkinson's and Alzheimer's all at the same time. The drug has a black box warning for risk of depression and suicidal thoughts.
Disease info: NIH Neurological Institute Huntington's Disease Information Page Huntington's Disease Society of America

ALS (amyotrophic lateral sclerosis- Lou Gerhig's Disease)

Patient population: 20,000 in the U.S. with 5,000 new diagnoses per year
Notable Patients: Stephen Hawking (physicist)
New Drug: Radicava (edaravone)
Company: Mitsubishi Tanabe Pharma America
Estimated Cost: $1,000 per infusion; about $146,000 per year
Administered: Intravenous infusion; dosing follows a 2-weeks on, 2-weeks off pattern which changes after first treatment cycle. Drug was approved in Japan in 2015 and the FDA engaged the drug developer to apply to sell it in the U.S. It was approved here based on a six-month Japanese clinical trial - in which 137 patients were randomized to receive either Radicava or placebo - company says Radicava reduced the decline in physical ability by 33 percent compared to placebo. This limited data shows possible serious side effects of hives, shortness of breath, bruising, and gait disturbance.
Disease info: NIH Neurological Institute Amyotrophic Lateral Sclerosis Information Page

Patient population estimates from Cost estimates from press releases and pharmacy web sites. Table of information researched and compiled by