Mirum’s CTEXLI™ (chenodiol) Tablets Receives FDA Approval for Treatment of Cerebrotendinous Xanthomatosis (CTX)
Mirum Pharmaceuticals (NASDAQ: MIRM) has received FDA approval for CTEXLI™ (chenodiol) tablets, becoming the first and only approved treatment for adults with cerebrotendinous xanthomatosis (CTX), a rare and progressive disease. The approval is based on the Phase 3 RESTORE study results, which demonstrated highly significant reduction in bile alcohols (p<0.0001) with a 20-fold difference between placebo and CTEXLI-treated patients.
The study showed CTEXLI improved both urine bile alcohol and serum cholestanol levels. In CTX patients, a deficiency of bile acid chenodeoxycholic acid leads to toxic accumulation of cholestanol, causing irreversible neurologic dysfunction. The drug has been granted FDA Orphan Drug exclusivity and will be available through Mirum Access Plus (MAP), the company's patient support program.
Mirum Pharmaceuticals (NASDAQ: MIRM) ha ricevuto l'approvazione della FDA per le compresse di CTEXLI™ (chenodiolo), diventando il primo e unico trattamento approvato per gli adulti affetti da xantomatosi cerebrotendinosa (CTX), una malattia rara e progressiva. L'approvazione si basa sui risultati dello studio di Fase 3 RESTORE, che ha dimostrato una riduzione altamente significativa degli alcoli biliari (p<0.0001) con una differenza venti volte superiore tra i pazienti trattati con CTEXLI e quelli in placebo.
Lo studio ha mostrato che CTEXLI ha migliorato sia i livelli di alcol biliari nelle urine che i livelli di colestanol sierico. Nei pazienti con CTX, una carenza dell'acido biliare chenodeossicolico porta ad un accumulo tossico di colestanol, causando disfunzione neurologica irreversibile. Il farmaco ha ricevuto l'esclusività come farmaco orfano dalla FDA e sarà disponibile attraverso Mirum Access Plus (MAP), il programma di supporto ai pazienti dell'azienda.
Mirum Pharmaceuticals (NASDAQ: MIRM) ha recibido la aprobación de la FDA para las tabletas de CTEXLI™ (chenodiol), convirtiéndose en el primer y único tratamiento aprobado para adultos con xantomatosis cerebrotendinosa (CTX), una enfermedad rara y progresiva. La aprobación se basa en los resultados del estudio de Fase 3 RESTORE, que demostró una reducción altamente significativa en los alcoholes biliares (p<0.0001) con una diferencia de 20 veces entre los pacientes tratados con CTEXLI y los del grupo placebo.
El estudio mostró que CTEXLI mejoró tanto los niveles de alcohol biliar en orina como los niveles de colestanol en suero. En los pacientes con CTX, una deficiencia del ácido biliar chenodeoxicólico conduce a una acumulación tóxica de colestanol, causando disfunción neurológica irreversible. El medicamento ha recibido exclusividad de medicamento huérfano de la FDA y estará disponible a través de Mirum Access Plus (MAP), el programa de apoyo a pacientes de la empresa.
미럼 제약 (NASDAQ: MIRM)은 CTEXLI™ (켄오디올) 정제에 대한 FDA 승인을 받았으며, 이는 성인에서 뇌힘줄 황색종 (CTX)에 대한 최초이자 유일한 승인 치료제가 되었습니다. 이 승인은 3상 RESTORE 연구 결과를 기반으로 하며, 이는 위약과 CTEXLI 치료를 받은 환자 간의 담즙 알코올 수치에서 20배의 차이를 보이며 (p<0.0001) 매우 유의미한 감소를 입증했습니다.
연구 결과 CTEXLI가 소변 담즙 알코올과 혈청 콜레스탄올 수치를 모두 개선한 것으로 나타났습니다. CTX 환자에서는 담즙산인 케노데옥시콜산의 결핍이 콜레스탄올의 독성 축적을 초래하여 되돌릴 수 없는 신경학적 기능 장애를 유발합니다. 이 약물은 FDA의 고아약 독점권을 부여받았으며, 회사의 환자 지원 프로그램인 Mirum Access Plus (MAP)를 통해 제공될 예정입니다.
Mirum Pharmaceuticals (NASDAQ: MIRM) a reçu l'approbation de la FDA pour les comprimés de CTEXLI™ (chenodiol), devenant ainsi le premier et unique traitement approuvé pour les adultes atteints de xanthomatose cérébrotendineuse (CTX), une maladie rare et progressive. L'approbation est basée sur les résultats de l'étude de phase 3 RESTORE, qui a montré une réduction hautement significative des alcools biliaires (p<0.0001) avec une différence de 20 fois entre les patients traités par CTEXLI et ceux sous placebo.
L'étude a montré que CTEXLI améliorait à la fois les niveaux d'alcools biliaires dans les urines et les niveaux de cholestanol sérique. Chez les patients atteints de CTX, une carence en acide biliaire, l'acide chenodeoxycholique, entraîne une accumulation toxique de cholestanol, provoquant des dysfonctionnements neurologiques irréversibles. Le médicament a reçu l'exclusivité de médicament orphelin de la FDA et sera disponible via Mirum Access Plus (MAP), le programme de soutien aux patients de l'entreprise.
Mirum Pharmaceuticals (NASDAQ: MIRM) hat die FDA-Zulassung für CTEXLI™ (Chenodiol) Tabletten erhalten und ist damit die erste und einzige zugelassene Behandlung für Erwachsene mit cerebrotendinöser Xanthomatose (CTX), einer seltenen und fortschreitenden Erkrankung. Die Zulassung basiert auf den Ergebnissen der Phase-3-Studie RESTORE, die eine hochsignifikante Reduktion der Gallensalze (p<0.0001) mit einem 20-fachen Unterschied zwischen Placebo- und CTEXLI-behandelten Patienten zeigte.
Die Studie zeigte, dass CTEXLI sowohl die Urin-Gallensalze als auch die Serum-Cholestanolspiegel verbesserte. Bei CTX-Patienten führt ein Mangel an Gallensäure, Chenodeoxycholsäure, zu einer toxischen Ansammlung von Cholestanol, die irreversible neurologische Dysfunktionen verursacht. Das Medikament erhielt von der FDA die Exklusivität als Orphan Drug und wird über Mirum Access Plus (MAP), das Patientenunterstützungsprogramm des Unternehmens, verfügbar sein.
- First and only FDA-approved treatment for CTX in adults
- Granted Orphan Drug exclusivity
- Phase 3 RESTORE study showed highly significant results (p<0.0001)
- Demonstrated 20-fold improvement vs placebo in primary endpoint
- None.
Insights
The FDA approval of CTEXLI marks a transformative moment for both Mirum Pharmaceuticals and the CTX treatment landscape. This approval carries significant commercial implications beyond its clinical importance:
The designation as the first and only approved treatment for CTX, combined with Orphan Drug exclusivity, provides Mirum with substantial market protection and pricing power. In the rare disease space, such exclusivity typically translates to premium pricing potential, particularly given the absence of approved alternatives.
The robust efficacy data from the RESTORE study, demonstrating a 20-fold difference in bile alcohol levels between treatment and placebo groups, provides strong clinical validation that could support favorable reimbursement discussions with payers. The establishment of Mirum Access Plus (MAP) as a dedicated patient support program demonstrates a comprehensive commercialization strategy, critical for rare disease treatments where patient identification and support are paramount.
From a market perspective, while CTX is ultra-rare, the approval positions Mirum as the definitive leader in this therapeutic area. The unmet medical need and potential to prevent irreversible neurological dysfunction creates a compelling value proposition. The successful development and approval of CTEXLI also validates Mirum's capabilities in rare disease drug development, potentially increasing investor confidence in their broader pipeline.
The approval's timing is particularly strategic as it enables Mirum to establish market presence and build relationships with specialists before potential competition emerges. This first-mover advantage, combined with orphan exclusivity, provides a significant window to optimize market penetration and establish CTEXLI as the standard of care in CTX treatment.
- CTEXLI is the first and only medication approved for the treatment of CTX in adults
- Approval based on Phase 3 RESTORE study results
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CTEXLI granted
U.S. FDA Orphan Drug exclusivity for the treatment of CTX
The approval is based on data from the Phase 3 RESTORE study evaluating the safety and efficacy of CTEXLI in adult patients with CTX by measurement of urine bile alcohols and other secondary measures. The primary endpoint of reduction in bile alcohols (urine 23S-pentol) was highly statistically significant (p<0.0001). At the end of the randomized double-blind withdrawal period, there was a 20-fold difference between placebo and CTEXLI treated patients in urine 23S-pentol levels.
In CTX, a deficiency of the bile acid chenodeoxycholic acid (CDCA) leads to a buildup of bile alcohols which precedes a toxic accumulation of cholestanol. Cholestanol is the key driver of symptomatic burden and disease progression, including irreversible neurologic dysfunction. Results from the RESTORE study demonstrated that treatment with CTEXLI not only improved urine bile alcohol levels but also serum cholestanol levels. Additionally, a greater proportion of patients receiving placebo required blinded rescue therapy, demonstrating the robustness of the effect.
“The FDA’s approval of CTEXLI is tremendous as it unlocks an opportunity to better identify and treat adult patients with CTX in
“Cerebrotendinous xanthomatosis (CTX) is a rare disease that can present with early cataracts, tendon lipid deposits, and significant neurologic disease, and the latter may be prevented with earlier diagnosis and treatment,” said Ernst J. Schaefer, MD, professor of medicine at Tufts University School of Medicine in
“CTX is a devastating disease that is often diagnosed in early adulthood with progressive symptoms that can have a significant impact on a person’s quality of life,” said Jean Pickford, executive director, CTX Alliance. “We are thrilled that CTEXLI is now approved and hope that patients with this disease are diagnosed earlier and can avoid potentially irremediable disease progression and many of the debilitating symptoms associated with CTX.”
CTEXLI will be available through Mirum Access Plus (MAP), Mirum’s patient support program. Patients currently on Mirum products or those with a prescription for CTEXLI can coordinate with MAP to receive fulfillment support by dialing (855) MRM-4YOU (855-676-4968) or visiting www.ctexli.com for more information.
About Cerebrotendinous Xanthomatosis
Cerebrotendinous xanthomatosis (CTX) is an autosomal, recessive, progressive genetic disorder resulting from a deficiency of a key enzyme in the bile acid synthesis pathway. CTX is characterized by fatty yellow nodules (xanthomas) located in the connective tissues within the brain. These deposits can cause progressive damage to the brain and other areas of the body. As the clinical course progresses, irreversible neurological deterioration leads to premature death. CTX is a rare disease affecting one to two thousand people in
About CTEXLI™ (chenodiol) tablets
CTEXLI™ (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries.
IMPORTANT SAFETY INFORMATION
CTEXLI can cause side effects, including:
Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching.
Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection.
Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines.
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets.
LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the
Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder.
To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X).
Forward-Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, the potential benefits of CTEXLI for appropriate patients, and the real life CTEXLI treated patients’ reduction of symptoms of CTX. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “will,” “could,” “can,” “would,” “potential,” “hope,” “opportunity,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum’s business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law. A further description of risks and uncertainties can be found in Mirum’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 and subsequent filings with the
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Media Contact:
Erin Murphy
media@mirumpharma.com
Investor Contact:
Andrew McKibben
ir@mirumpharma.com
Source: Mirum Pharmaceuticals, Inc.
FAQ
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