The pharmaceutical company Biohaven has announced that they will be using an expanded access program to make an experimental drug for amyotrophic lateral sclerosis (ALS) available to patients before F.D.A. approval of the drug. The full story can be read here, at ParmaVoice.
ALS, also known as motor neurone disease or Lou Gehrig’s disease, is a life-shortening condition that affects the brain and nerves and is associated with progressive muscle weakening. Most people who are affected are in their 60s and 70s. The disease usually progresses gradually, and early symptoms may include leg or ankle weakness, slurred speech, weight loss, a weakened grip, and muscle cramps. People often report experiences such as difficulty walking up and down stairs, or opening jars. There is currently no cure for ALS, and treatment focuses on reducing symptoms, prolonging lifespan, and increasing a patient’s quality of life. Those with the condition may be offered physiotherapy to improve muscle strength, appointments with speech and diet experts, medicines to improve swallowing and reduce muscle stiffness, and a drug called riluzole that slows the condition’s progression.
The drug under development by the company Biohaven is called BHV-0223 and has the active ingredient riluzole. It is a tablet that should be taken twice per day; 40mg of it provides equivalent effects to 50mg of Rilutek (riluzole). This may help patients because riluzole can harm the liver, but because BHV-0223 is given in a lower dosage it is expected to have fewer negative effects on the liver. Furthermore, BHV-0223 has been formulated for sublingual (under the tongue) administration. It will quickly dissolve in a patient’s mouth. This is likely to help patients who have trouble swallowing – a problem that affects approximately one-third of ALS patients at the time of diagnosis, and higher proportions as the disease progresses.
BHV-0223 is still in the process of working towards FDA approval to make the drug widely available to ALS patients. However, Biohaven has been granted permission to establish an expanded access program (EAP) for it. The program will allow medical professionals to provide BHV-0223 to eligible patients for free. EAPs are FDA-regulated programs that allow certain medicines, which have not yet achieved approval, to be distributed to some patients in the case of serious or life-threatening diseases that do not have equivalent medicines already available. More information about the EAP for BVH-0223, including the eligibility criteria, is available here.
Calaneet Balas, the president and CEO of The ALS Association, says
“It is great for the ALS community that Biohaven is taking advantage of the FDA’s expanded access program.”
