The move started the celebration in the LEMS community and fueled the warehouse Catalyst Pharmaceuticals, producer of the $ 375,000 drug, called Firdapse. The company's share price has fallen about 50% since Monday.
The FDA said it was approved by Ruzurgi – made by a small company called Jacobus Pharmaceutical Co. – for children with LEMS aged 6 to 17 years.
We continue to be committed to facilitating the development and approval of rare disease treatments, especially in children, says Dr. Billy Dunn, Director of Division of Neurology Products at the FDA's Drug Investigation and Research Center.
"This approval will provide a need for treatment options for children with LEMS that have significant weakness and fatigue which can often cause major difficulties with daily activities."
Although Ruzurgi was approved for children, the FDA decision made it possible for adults with LEMS to get the drug off-label if their treating physician believes it will help them.
"In general, if a drug is FDA approved, it can be used off-label as long as doctors feel it is indicated to the patient," said Dr. Vincent Rajkumar, a hematologist oncologist with the Mayo Clinic. "It's how drugs that are approved for a disease can be used for almost everyone else."
Rajkumar said he hopes that Jacobus makes the drug cheaper than following Catalyst's leadership.
Jacobus has not announced a prize for Ruzurgi, but owner Laura Jacobus said the company wants to ensure that the drug is affordable to patients.
"We have long been working on this drug and have invested considerable resources," she said in a written statement. "Although we have not created a prize for Ruzurgi, it is still our goal to ensure that Ruzurgi is affordable and accessible to children living with LEMS."
The FDA said it is not known how many LEMS patients it is, but Jacobus estimates it is less than 15. According to the National Organization for Rare Disorders, LEMS affects about 400 people in the United States. The FDA said a total of 3 people out of 1 million worldwide LEMS.
Last year, FDA approved Catalysts Firdapse for adults. When the company announced a $ 375,000 price tag, it was upset.
Sen. Bernie Sanders of Vermont, a democratically hopeful, sent a blowing letter to the company, demanding answers and calling it "corporate greed" at its worst. He also asked the FDA to allow Jacobus – who had made his pre-authorization drug available for many years at no cost to patients through a compassionate utility program – to continue doing the medicine.
a statement Wednesday , Sanders celebrated the FDA's decision, saying that no patient "should worry about whether they will suffer or die to increase the indecent profits of a pharmaceutical company."
"This week marks a victory for patients with LEMS and American taxpayers, and a blow to growing by Big Pharma," he said. "Suffer from patients who have received life-saving drugs for free from Jacobus Pharmaceuticals – to a competitor, Catalyst, bought the rights to the drug, received the market exclusivity, and began charging $ 375,000 a year to those in desperate need.
" me about the news that Jacobus Pharmaceuticals has now received approval for their product to treat childhood LEMS. I urge Catalyst to respond by lowering its own outrageous prices. If not, Congress must act. »
Krishan Patel, whose mother has LEMS, said the news was a relief.
"The adult LEMS community is excited and trying to move forward to get the new drug off-label," he said. "We've asked my mom's doctor to prescribe the drug as an off-label drug, And then we have to go through the insurance process. "
His mother, Bhanu Patel, added that she was" overwhelmed by the news. "She told CNN earlier that she had ranting Catalysts drugs because of her high price.
" Jacobus Pharmaceuticals has taken care of so many LEMS patients over the years through their compassionate utility program, and they truly deserved this approval, she said.
David Schull, catalyst spokesman, says the company is still "focused on continuing the launch of Firdapse to get this drug to patients in need, including the many who did not have access to any treatment prior to the FDA approval of the drug.  "The catalyst will try a detailed launch update on Monday as part of the financial result," he said.