2,400 people remain without rare disease drug as Takeda manufacturing woes continue
Since September 2019, 2,400 people with the rare condition known as hypoparathyroidism have been waiting for any sign that supplies of their reliable injection from Takeda might reemerge.
Last Wednesday, however, Takeda announced that supplies won’t be ready for at least another year, stranding those who still can’t access the drug, known as Natpara (parathyroid hormone), via the company’s Special Use Program (SUP), which is only for about 400 of those who are at extreme risk of life-threatening complications.
“At this time we do not expect a return to market before March 31, 2022,” Takeda said in a letter.
The difficulties with hypoparathyroidism occur due to low levels of calcium in the blood. Symptoms can range from the more mild tingling or numbness in the fingers and toes, to severe muscle cramps and spasms, as well as breathing issues that can lead to hospitalization, according to the National Organization for Rare Disorders.
Even for those who still have access to Natpara via Takeda’s special program, last week’s announcement was a difficult one.
“The announcement was definitely a devastating one for the entire hypopara community, especially after the virtual hypopara conference in October, where Takeda had remarked that they hoped to announce their plans to return to market in March,” Heather Novak, a Detroit-based romance novelist who is on the SUP but told Endpoints News via email that she also deals with intermittent supply issues.
In its update, Takeda offered few specifics on what’s causing the manufacturing issues at its contractor plants that are overseen by Takeda. Back in 2019, FDA announced the Class I (the highest degree of potential health hazard) recall for Natpara, and said it relates to rubber stopper particles clogging the needle and leading to underdosing, according to letters sent to patients.
In its update, Takeda told patients last week, “While we have made progress on the original issue that led to the U.S. recall, which was the issue of rubber particulates originating from the rubber septum of the NATPARA cartridge, we have not yet reached a resolution.”
Another manufacturing delay, which Takeda confirmed is separate from the original issue in 2019, is also affecting supplies the 100-mcg version of Natpara under the SUP. Novak explained that when there’s an expected shortage of one one or more strengths of Natpara, like the 100-mcg version, Takeda works with doctors to switch prescriptions or dosing for patients to prevent anyone on the SUP from going without it.
Takeda acquired Natpara when it bought out Shire for $62 billion in early 2019. In 2018, the last full year of Natpara sales, the treatment brought in about $230 million. Since the Natpara recall in September 2019, no US revenue has been recorded and the concerns are growing.
“We’ve been told that they plan to keep the SUP going until the drug returns to market. Many of us, of course, are wondering IF it will return to the US market,” Novak said.
Those who haven’t gained access to the SUP are struggling even more with the Takeda delays.
Rita McCullough of Le Center, MN, told Endpoints via email that within a week of the 2019 recall, she ended up in the emergency department twice. She said the condition affects her breathing and she has severe muscle spasms without Natpara.
“I was crushed that I had to go back to all the pills and the alarm every two hours just so I can breathe,” she said, noting that Natpara did work for her. “The form letters Takeda releases are pointless. I want to know what’s going on with the rubber particulates.”
Bob Sanders, chairman of the board of the nonprofit Hypoparathyroidism Association, said he isn’t aware of anyone who has died from a lack of access to Natpara. He said some who have lost access to Natpara are taking Eli Lilly’s Forteo off-label, while others are on calcium supplements and Calcitriol, which is not always approved for purchase by insurance companies and can create financial burdens for some patients.
“We all hope that there is no calcium crash as most hospitals lack the understanding to treat a hypopara patient because it is so rare,” Sanders told Endpoints News via email. “Another year without Natpara represents another year of symptoms and struggles.”
He added that his association has a listening session scheduled with the FDA and he hopes to get a patient-focused drug development meeting scheduled for next year “to talk about our concerns and how the agency can best help the patient community.”