It has been one year since Mitsubishi Tanabe Pharma America’s (MTPA) Radicava was approved as the first treatment for amyotrophic lateral sclerosis (ALS) in more than 20 years. Since its approval, MTPA has been aggressive in its efforts to get the medication in the hands of ALS patients. The drug officially rolled out to U.S. patients in August 2017.
As of April, about 2,500 patients in the United States have received treatment with the newly approved therapeutic. Also called Lou Gehrig’s disease, ALS is a rare disease that attacks and kills the nerve cells that control voluntary muscles. The Centers for Disease Control and Prevention estimates that approximately 12,000 to 15,000 Americans have ALS, with about 5,000 to 6,000 diagnosed annually. The average life expectancy of an ALS patient is approximately three to five years after diagnosis and only 10 percent of patients survive for more than 10 years. Death usually is a result of respiratory failure.
Radicava is not a cure for ALS, but it slows decline in the daily functioning of ALS patients. The drug was approved based on Phase III clinical trials that demonstrated patients taking the drug had a slower decline in physical function than those taking a placebo. Data demonstrated patients who received Radicava for six months experienced significantly less decline in physical function — by 33 percent.
Kevin O’Brien, MTPA’s vice president of market access, told BioSpace that the rollout of Radicava has been a bit slow, primarily due to hurdles with insurance. Another issue the company has diligently worked on since the FDA approval is the establishment of dosing centers or approval for in-home infusions, O’Brien said as ALS Awareness Month came to an end.
“For a lot of patients finding site of care was difficult. Now there are more than 1,200 infusion centers across the U.S. We’ve tried to hit every hot spot across the country,” O’Brien said.
O’Brien added that the company is anticipating opening more infusion centers to assist ALS patients with their treatments. The company is also working with the Veterans Administration to help the men and women in the armed forces who develop ALS. There are an estimated 3,600 ALS patients in the military. Research has shown that those who serve in the military, regardless of branch or time of service, are more susceptible to the disease. Yet, no one knows why. But, O’Brien said MTPA is doing all it can to ensure those patients have access to Radicava.
Not only is O’Brien focused on increasing access to Radicava, he said the goal over the next six months is to reduce the time it takes between the diagnosis of ALS and the first infusion of the therapy. O’Brien said the goal is for the time frame to be about 30 days, which would be a significant reduction from the more than two months it usually takes. Since they undertook the mission, O’Brien said they have been able to reduce the time it takes for treatment to less than 40 days.
“We’re doing all we can to help the patients. We want to get them diagnosed and into therapy as quickly as possible,” he said. “Since the launch of Radicava it’s been an evolution of learning.”
For O’Brien and the rest of the MTPA family, their days revolve around helping patients. Debbie Etchison, head of public affairs for MTPA, said employees at the company come to work with a sense of purpose each day, especially since the approval of Radicava.
“It makes getting out of bed every day an easy thing because everything we do is focused on the patient,” Etchison said. O’Brien agreed, adding the company measures everything in patients. O’Brien said in his annual review earlier this month he was not asked how he had helped the company achieve its goals, but rather his superiors wanted to know how many patients he had been able to help and how that number could be increased over the next year. He noted that he and other executives often meet with ALS patients to ascertain how they can improve point of care among their target patient population.
“That’s how we live here. We make sure we’re accessible. We’re about patients and how to help them. We’re not talking about the drug, we’re talking about the patients,” he said. “ALS, it’s one of those diseases, the diagnosis has to be the worst thing you can ever get in your life. The disease just takes everything away from these folks.”
While the company continues to improve patient access for Radicava, Etchison said MTPA is continuing to invest in developing treatments for ALS, with hopes for an eventual cure. In April, MTPA’s parent company Mitsubishi Tanabe Pharma Corporation worked in partnership with Amgen to back the launch of Cambridge, Mass.-based QurAlis, which is developing three therapeutics targeting subsets of ALS. QurAlis is currently developing therapies for three different forms of ALS with known disease mechanisms which include: a transformative device to remove toxic proteins; a drug that mediates overactive neurons and prevents them from dying; and a drug that restores a dysfunctional waste clearance system in cells. Success with any of these therapies will have a tremendous positive impact on ALS patients and their families, the company said when it launched.
“Our commitment to the community goes above and beyond Radicava. We’re investing in the search for a cure,” Etchison said.