Takeda vs. FDA
In the meantime, Takeda says it has been in regular contact with the FDA to try and work out how to bring Natpara back to U.S. patients who require it for symptom control.
Current outstanding issues between the company and FDA involve considerations related to dose accuracy, safety, and supply continuity, which the company hopes will be resolved in a timely manner.
Furthermore, shortly after the recall order, the company created a special use program that continues to support patients previously prescribed Natpara who would otherwise be at extreme risk of serious complications if forced to stop taking the drug.
“Originally intended for an extremely limited number of patients, the special use program has now enrolled approximately 300 patients,” Cheryl Schwartz, head, U.S. Hematology & Rare Business Unit, Takeda, said in a letter to members of the U.S. Hypoparathyroidism Association on Nov. 21.
The number is higher than expected and reflects the significant treatment needs of patients with serious disease, Ms. Schwartz noted.
“I want to reiterate that we do understand and sincerely regret the impact the Natpara recall is having on patients. We will provide another update as soon as we have more information to share. We continue to work around the clock to find ways to bring Natpara back to the broader hypoparathyroidism community,” she added.
“Patient safety always has been, and continues to be, the highest priority for Takeda,” she said.
Final efforts
Dr. Khan is currently involved in clinical trials evaluating Natpara in Canada (where it is not yet approved).
She was also involved in some of the pivotal studies that helped gain support of the product in the United States and European Union, including REPLACE, which showed that the injectable PTH molecule maintained serum calcium while reducing or eliminating requirements for calcium and active vitamin D supplementation.
“We now have 8 years of experience with this molecule, so we know that it’s safe and effective,” Dr. Khan emphasized.
Health Canada has not deemed Natpara problematic enough to stop the ongoing research program into injectable PTH therapies there, which is being headed up by Dr. Khan at McMaster University.
“The company has not yet applied for approval here, but when they do apply, I am sure they will have fixed this problem by then,” Dr. Khan said. “And I expect it will be fixed momentarily in the United States, where patients have been most affected.”
Asked if she was looking forward to getting Natpara back on track, Ms. Astolfi said “absolutely” several times.
“This recall has really had a large impact on our community,” she stressed. “And while it’s good that critically ill patients have access to the drug through the special-use program, we want to get everyone back to their best life and feeling great.”
A version of this story originally appeared on Medscape.com.