Contract development and manufacturing organization Catalent has been a major name in COVID-19 vaccine production, helping out Johnson & Johnson and Moderna. However, because of Operation Warp Speed, it also had to take away fill-finish capacity budgeted for Horizon Therapeutic's Tepezza (teprotumumuab-trbw), the only drug approved by the US Food and Drug Administration (FDA) to treat thyroid eye disease (TED).
While the drug shortage was resolved this week—neatly in time with Horizon's Q1 prediction—approximately 2,000 patients who were taking Tepezza during the third quarter of 2020 may have had to forgo the drug for more than 4 months.
Rare disease, one treatment
TED affects 16 per 100,000 women and 2.9 per 100,000 men in the general population, according to the National Organization for Rare Disorders. Common symptoms include extreme eye dryness, double vision, and bulging eyes, all of which Minnesota resident Vicki Grant experienced before starting Tepezza in April 2020.
"It did wonders right away; it really did," she says. She was able to get through six of eight doses, spaced 3 weeks apart, before she found out about the shortage, first from her TED-related Facebook groups and then from her ophthalmologist.
Off-label drugs like steroids or even the cancer drug rituximab have been used to treat TED before Tepezza's approval in January 2020, but Grant declined to start any new options during the temporary shortage. The side effects, both physical and mental, seemed too risky, and her symptoms weren't bad enough to warrant what would most likely be only temporary alleviation.
Instead, Grant waited, emailed people at the FDA to advocate return of Tepezza production, and stayed in close contact with her ophthalmologist and endocrinologist. She also continued taking her Graves' disease prescription, which she had hoped would end with her completion of the Tepezza regimen.
By the end of March, Grant's vision was beginning to blur again (meaning no more freeway driving), and the occasional headaches had come back along with slight bulging of her eyes. And although she was quick to say her symptoms weren't as bad as before, her ophthalmologist's estimates of Tepezza's return in May felt like a long way away.
"I feel bad for all those who are like I was [before Tepezza]," she said at the time, reflecting on her Facebook updates about people undergoing surgery to fix issues like bulging or misaligned eyes, double vision, retracted eyelids, and scarring. "When I see those pictures of people on Facebook, I can't help but think, 'God, I wish they'd start this up again.'"
Resupply to begin this month
As articles by Ophthalmology Times, Healio, and the Financial Times popped up about the shortage, Horizon submitted an FDA application for prior approval to increase the scale production of Tepezza. On Mar 30, it was approved, and the company announced that resupply would begin this month.
In the press release, Horizon advised patients to contact their infusion center directly or contact the Horizon Patient Access Liaison. Because Tepezza requires pre-authorization to be administered, people may also have to make sure those approvals have not lapsed during the interim, Terry Smith, MD, one of Tepezza's developers and the Graves' Disease & Thyroid Foundation's chief medical and scientific officer, told CIDRAP News.
"Our patients and we as physicians look forward to having the drug in our armamentarium again," he says. "Obviously it was not an ideal situation to have this disruption, but the public health situation with the pandemic demanded some actions by the FDA, and certainly the wisdom of that is understandable."
Tepezza's drug substance manufacturing is done at AGC Biologics, and drug product manufacturing is done at Catalent, but Horizon's chief communications officer Geoff Curtis says the company is also working on securing FDA approval to produce Tepezza at a second drug product manufacturer by the end of the year.