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FDA 重新考慮短缺問題，複合藥房可以恢複生產 tirzepatide
https://www.nbcnews.com/health/health-news/compounding-pharmacies-can-resume-making-tirzepatide-fda-reconsiders-s-rcna174551
Berkeley Lovelace Jr. 2024 年 10 月 14 日
FDA 的舉措是對複合貿易集團的訴訟的回應，該集團聲稱 tirzepatide（Zebpound 中的成分）仍然供不應求。
美國食品和藥物管理局在周五晚些時候提交的一份法庭文件中表示，它將允許藥劑師繼續生產複合版 tirzepatide（禮來糖尿病和減肥藥物 Mounjaro 和 Zepbound 中的活性成分），同時重新考慮將該藥物從全國短缺名單中刪除的決定。
這一意外舉措對配藥藥劑師和患者而言是一次重大勝利，他們在 10 月 2 日 FDA 宣布替澤帕肽短缺問題已解決後對 FDA 感到憤怒。
該藥物以及 Ozempic 和 Wegovy 中的活性成分索馬魯肽的短缺推動了配藥藥房生產自己版本的藥物的空前需求，患者表示這些藥物通常比品牌藥物更便宜且更容易獲得。
在 FDA 宣布短缺期間，配藥藥房生產“基本上是”短缺品牌藥物的仿製品是合法的，盡管製藥商強烈反對這種做法的合法性。
FDA 於 10 月 2 日宣布短缺結束，這意味著配藥藥劑師不得不停止配製替澤帕肽處方。 FDA 當時表示，生產大批量藥物的藥店將不再接受新的 tirzepatide 訂單，並有 60 天的時間來填寫現有訂單。
10 月 7 日，複合貿易組織外包設施協會起訴 FDA，聲稱該藥物仍然供應不足，因此應繼續留在短缺名單上。
FDA 周五提交的文件是對該組織的訴訟的回應，稱其舉動“實際上是原告在其動議中尋求的救濟”。該機構在文件中表示，目前，它不會對製造複合藥物的原告及其成員“采取行動”，因為它正在重新評估其決定。
2023 年 6 月，南卡羅來納州斯帕坦堡的 50 歲 Simone Williams 在她的 Mounjaro 儲蓄卡過期後被開了複方 tirzepatide。Simone Williams
南卡羅來納州斯帕坦堡的 50 歲 Simone Williams 表示，她對 FDA 將 tirzepatide 從短缺名單中剔除的決定深感不安。
去年，在她的 Mounjaro 儲蓄卡過期後，Williams 開始服用複方 tirzepatide 減肥。她負擔不起每月 1,060 美元的品牌藥自付費用。
Williams 需要“玩轉”並開具仍然短缺的複方 semaglutide 處方。
Williams 說，FDA 的更新是“好消息”，但她仍然很謹慎。
“在 FDA 做出明確決定之前，我仍然會對此感到緊張和憤怒，因為他們可能會回來說‘不，沒有短缺’，我又回到了原點，”她說。
tirzepatide 真的短缺嗎？
Tirzepatide 近兩年來一直在 FDA 的藥物短缺名單上。最初以 Mounjaro 的名稱批準用於治療 2 型糖尿病，許多患者被處方使用 tirzepatide 來減肥。去年，當它以 Zepbound 的名稱正式獲批用於減肥時，它的人氣飆升，加劇了短缺。
禮來已采取措施提高 tirzepatide 的產量，包括投入數十億美元建造一個專門生產更多藥物的新製造工廠。
美國衛生係統藥劑師協會 (American Society of Health-System Pharmacists) 藥房實踐和質量高級主管邁克爾·加尼奧 (Michael Ganio) 表示，該組織仍在聽取患者和護理人員的報告，稱提澤帕肽仍然難以獲得。該協會是一個跟蹤美國藥品短缺情況的組織。
美國衛生係統藥劑師協會仍將提澤帕肽列為短缺藥品，不過加尼奧表示，隨著藥劑師了解到有庫存，這種情況可能會改變。
“這種情況可能隻需要幾周時間就能實現供應平衡，”他說。“配送中心會得到補貨，然後將其發送到藥店。藥店可能都有積壓訂單，他們的客戶可能一直在從配藥藥房購買，現在正試圖從零售藥店和社區藥店購買。”
一些患者現在轉向流行減肥藥的仿製品
03:30
在北卡羅來納州卡裏經營減肥診所的胃腸病學家克裏斯托弗·麥高文博士表示，他認為訴訟與藥物是否真的短缺無關，而與複合 GLP-1 藥物的利潤豐厚性質有關。
“這是為了錢，”麥高文說。“複合行業將爭辯
供應鏈還不夠穩定，但實際上有數百萬人處於危險之中。這就是問題所在。”
禮來表示，tirzepatide 並不短缺。
禮來發言人 Jared Shapiro 在周一的一份聲明中表示，所有劑量的 Mounjaro 和 Zepbound 都是可用的，並警告說，重要的是患者“不要接觸未經測試、未經批準的仿製品的風險”。
“沒有什麽能改變這樣一個事實，即正如 FDA 所認識到的，Mounjaro 和 Zepbound 是可用的，短缺問題仍然‘得到解決’，”Shapiro 說。
FDA 沒有回應對其逆轉的更多評論請求。
FDA 發言人在周五晚上提交文件之前的一份聲明中表示，該機構理解患者對其宣布結束短缺的決定感到沮喪，並表示，“我們理解高藥價對患者有直接影響。”
“太多美國人買不起他們需要的藥物，”發言人說。“然而，FDA 沒有法律權力調查或控製製造商、分銷商和零售商設定的價格。”
發言人說，FDA 的配藥計劃旨在為需要這些藥物的患者保留獲得“合法銷售的配藥”的機會，盡管該機構通常建議患者在可用時使用 FDA 批準的藥物。
“安心”
與此同時，患者和配藥藥劑師正在慶祝 FDA 允許繼續配製替澤帕肽的決定。
提起訴訟的外包設施協會發言人在一份聲明中表示，“對我們的成員和他們服務的眾多患者來說，這令人鬆了一口氣。”
“我們認為，鑒於該機構在承認‘供應中斷’的情況下草率決定將該藥物從名單中剔除，這是一個公平的解決方案，”發言人說。 “最重要的是，如果 FDA 在短缺問題仍然存在的情況下重複其撤訴決定，我們將再次訴諸法庭。”
另一個行業組織藥房配藥聯盟在一份聲明中稱，這是外包設施協會的“非凡勝利”。
本月初，APC 與記者進行了電話會議，稱 FDA 的初步決定讓配藥藥房“措手不及”，讓患者“措手不及”。
禮來公司在 8 月首次表示，tirzepatide 短缺問題將很快得到解決，比 FDA 的正式決定早兩個月。
APC 上周末在給客戶的電子郵件中表示，它正在與律師合作，以弄清 FDA 的新申請是否意味著它不會對所有生產 tirzepatide 的配藥藥房采取執法行動，還是隻對訴訟中的原告采取執法行動。
“我們的目標是以書麵形式讓 FDA 對所有目前配製替澤帕肽注射劑的藥房采取執法自由裁量權，”APC 在電子郵件中寫道。
對於北卡羅來納州格雷厄姆市 59 歲的伊麗莎白·肯利來說，FDA 的決定讓她“安心”。
肯利在 3 月份因 Wegovy 短缺而難以找到後，醫生給她開了一種替澤帕肽的複合版本。
上周，肯利的配藥藥劑師通知她，由於替澤帕肽不再在短缺名單上，他們需要停止生產複合版本或尋找其他供應商。藥劑師表示，他們將分發剩餘的庫存，並希望在年底前繼續為患者提供處方。
由於 FDA 周五提交了申請，肯利不需要“匆忙製定 B 計劃”。
自從她開始服用這種複方藥物以來，她說她已經減掉了 30 磅，並希望再減掉 25 磅。
“複方藥房是我繼續接受這種對我非常成功的治療的生命線，”肯利說。“我明白研發公司需要賺錢，但他們也需要與保險公司合作，確保公平公正地獲得這些藥物。”
伯克利·洛夫萊斯二世
伯克利·洛夫萊斯二世是 NBC 新聞的健康和醫療記者。他報道食品和藥物管理局，特別關注新冠疫苗、處方藥定價和醫療保健。他之前曾在 CNBC 報道過生物技術和製藥行業。
Compounding pharmacies can resume making tirzepatide as FDA reconsiders shortage
The Food and Drug Administration said in a court filing late Friday that it would allow pharmacists to continue making compounded versions of tirzepatide — the active ingredient in Eli Lilly’s diabetes and weight loss drugs Mounjaro and Zepbound — while it reconsiders its decision to remove the drug from its nationwide shortage list.
The surprise move is a major victory for compounding pharmacists and patients who were furious with the FDA after its announcement on Oct. 2 that the tirzepatide shortage was resolved.
Shortages of the drug, along with semaglutide — the active ingredient in Ozempic and Wegovy — have fueled unprecedented demand for compounding pharmacies to make their own versions of the medicines, which patients say are often cheaper and easier to come by than the brand-name versions.
During FDA-declared shortages, it’s legal for compounding pharmacies to make versions that are “essentially a copy” of the brand-name drugs in shortage, though drugmakers have vehemently pushed back against the legitimacy of this practice.
The FDA’s announcement on Oct. 2 declaring the shortage over meant that compounding pharmacists had to stop filling tirzepatide prescriptions. It said at the time that pharmacies that produce large batches of medications would no longer be able to accept new orders of tirzepatide and had 60 days to fill their existing orders.
On Oct. 7, the Outsourcing Facilities Association, a compounding trade group, sued the FDA, claiming that the drug is still in short supply and should therefore remain on the shortage list.
The FDA’s filing Friday was in response to the group’s suit, saying its move was “effectively the relief that Plaintiffs sought in their motion.” In the filing, the agency said that, for the time being, it wouldn’t “take action” against the plaintiffs and their members making compounded versions of the drugs as it re-evaluates its decision.
Simone Williams, 50, of Spartanburg, S.C., was prescribed compounded tirzepatide in June 2023 after her Mounjaro savings card expired.Simone Williams
Simone Williams, 50, of Spartanburg, South Carolina, said she had been deeply upset with the FDA’s decision to take tirzepatide off the shortage list.
Williams began taking compounded tirzepatide for weight loss last year after her Mounjaro savings card expired. She couldn’t afford the $1,060 a month out of pocket for the brand-name drug.
Williams would need to “play switcheroo” and get a prescription for compounded semaglutide, which remains in shortage, instead.
The FDA’s update is “good news,” Williams said, but she’s still cautious.
“Until the FDA makes a solid decision, I’m still going to be nervous and up in arms about it because they could come back and say, ‘no, there’s not a shortage’ and I’m back at square one,” she said.
Is tirzepatide really in shortage?
Tirzepatide was on the FDA’s drug shortage list for nearly two years. Initially approved under the name Mounjaro to treat Type 2 diabetes, many patients were prescribed tirzepatide off-label for weight loss. When it was officially approved for weight loss last year — under the name Zepbound — it soared in popularity, exacerbating the shortages.
Lilly has taken steps to ramp up production of tirzepatide, including committing billions to a new manufacturing facility dedicated to producing more of the drug.
Michael Ganio, senior director of pharmacy practice and quality at the American Society of Health-System Pharmacists, a group that tracks drug shortages in the U.S., said the group is still hearing reports from patients and caregivers suggesting tirzepatide continues to be hard to come by.
The ASHP still lists tirzepatide as in shortage, although Ganio said that could change as pharmacists learn that there is supply in stock.
“This can be the kind of thing that just takes a couple of weeks for supply to even out,” he said. “Distribution centers get replenished and then they send it out to pharmacies. The pharmacies probably all have back orders and they have customers that maybe have been getting it from the compounding pharmacy and are now trying to get it from a retail pharmacy and community pharmacy.”
Dr. Christopher McGowan, a gastroenterologist who runs a weight loss clinic in Cary, North Carolina, said he believes the lawsuit is less about whether the drug is genuinely in shortage and more about the lucrative nature of compounded GLP-1 medications.
“It’s about money,” McGowan said. “The compounding industry will argue that the supply chain isn’t stable enough, yet, but really there are many millions at stake. That’s the issue.”
Lilly says that tirzepatide is not in shortage.
In a statement Monday, Jared Shapiro, a Lilly spokesperson, said all doses of Mounjaro and Zepbound are available, warning that it’s important that patients “not be exposed to the risks in taking untested, unapproved knockoffs.”
“Nothing changes the fact that, as FDA has recognized, Mounjaro and Zepbound are available and the shortage remains ‘resolved,’” Shapiro said.
The FDA didn’t respond to a request for additional comment on its reversal.
An FDA spokesperson said in a statement prior to the Friday evening filing that the agency understands the patients’ frustration about its decision to declare an end to the shortage, saying, “we understand that high drug prices have a direct impact on patients.”
“Too many Americans are priced out of the medicines they need,” the spokesperson said. “However, the FDA has no legal authority to investigate or control the prices set by manufacturers, distributors and retailers.”
The FDA’s compounding program aims to preserve access to “lawfully marketed compounded drugs” for patients who need them, the spokesperson said, although the agency generally recommends patients use FDA-approved drugs when available.
‘Peace of mind’
Meanwhile, patients and compounding pharmacists are celebrating the FDA decision to allow tirzepatide compounding to continue.
A spokesperson for the Outsourcing Facilities Association, which filed the lawsuit, said in a statement that it was “relieved, for our members and the many patients that they serve.”
“We believe that this is a fair resolution in light of the agency’s rash decision to take the drug off the list at a time when the agency has acknowledged ‘supply disruptions,’” the spokesperson said. “Most important, should the FDA repeat its removal decision when a shortage still genuinely exists, we will return to court.”
In a statement, the Alliance for Pharmacy Compounding, another industry group, called it an “an extraordinary win” for the Outsourcing Facilities Association.
APC held a call with reporters earlier this month, saying that the FDA’s initial decision had caught compounding pharmacies “off guard” and left patients “flat-footed.”
Lilly had first indicated in August that the tirzepatide shortage would be resolved soon, two months before the official FDA decision.
The APC said in an email to clients over the weekend that it’s working with lawyers to get clarity on whether the FDA’s new filing means that it will not take enforcement against all compounding pharmacies who make tirzepatide or just the plaintiffs in the lawsuit.
“Our aim is to get in writing that FDA will take enforcement discretion related to all pharmacies compounding copies of tirzepatide injection at this time,” the APC wrote in the email.
For Elizabeth Kenly, 59, of Graham, North Carolina, the FDA’s decision gives her “peace of mind.”
Kenly was prescribed a compounded version of tirzepatide in March after she had trouble finding Wegovy because it was in shortage.
Last week, Kenly’s compounding pharmacists informed her that, because tirzepatide was no longer on the shortage list, they would need to stop making the compounded versions or seek out a different supplier. The pharmacists said that they would dispense the remaining stock and hoped to continue providing patients with prescriptions through the end of the year.
Because of the FDA’s filing Friday, Kenly doesn’t need to “scramble to figure out a plan B.”
Since she started the compounded drug, she says she has lost 30 pounds and wants to lose another 25.
“The compounding pharmacy is my lifeline to continue a therapy that has been so successful for me,” Kenly said. “I understand that research and development companies need to make money, but they also need to work together with insurance companies to ensure fair and equitable access to these medications.”
Berkeley Lovelace Jr. is a health and medical reporter for NBC News. He covers the Food and Drug Administration, with a special focus on Covid vaccines, prescription drug pricing and health care. He previously covered the biotech and pharmaceutical industry with CNBC.
