Volixibat Granted Breakthrough Therapy Designation for Cholestatic Pruritus in Primary Biliary Cholangitis
Mirum Pharmaceuticals (Nasdaq: MIRM) announced that the FDA has granted Breakthrough Therapy Designation to volixibat for treating cholestatic pruritus in patients with primary biliary cholangitis (PBC). This designation is based on positive interim results from the Phase 2b VANTAGE study, which showed statistically significant improvement in pruritus compared to placebo. The study's confirmatory portion is ongoing, with enrollment expected to complete in 2026.
The Breakthrough Therapy Designation aims to expedite the development and review of drugs for serious diseases, requiring preliminary clinical evidence of substantial improvement over existing therapies. Mirum's chief medical officer, Dr. Joanne Quan, emphasized the importance of addressing PBC-related itch and the company's commitment to making volixibat available to patients as quickly as possible.
Mirum Pharmaceuticals (Nasdaq: MIRM) ha annunciato che la FDA ha concesso la Designazione di Terapia Innovativa a volixibat per il trattamento del prurito colestatico nei pazienti affetti da colangite biliare primitiva (PBC). Questa designazione si basa sui risultati intermedi positivi dello studio di Fase 2b VANTAGE, che ha mostrato un miglioramento statisticamente significativo del prurito rispetto al placebo. La parte di conferma dello studio è attualmente in corso, con l'arruolamento previsto per completarsi nel 2026.
La Designazione di Terapia Innovativa mira ad accelerare lo sviluppo e la revisione dei farmaci per malattie gravi, richiedendo evidenze cliniche preliminari di un miglioramento sostanziale rispetto alle terapie esistenti. Il direttore medico di Mirum, Dr. Joanne Quan, ha sottolineato l'importanza di affrontare il prurito legato alla PBC e l'impegno dell'azienda a rendere volixibat disponibile ai pazienti nel più breve tempo possibile.
Mirum Pharmaceuticals (Nasdaq: MIRM) anunció que la FDA ha otorgado la Designación de Terapia Innovadora a volixibat para el tratamiento del prurito colestático en pacientes con colangitis biliar primaria (PBC). Esta designación se basa en resultados interinos positivos del estudio de Fase 2b VANTAGE, que mostró una mejora estadísticamente significativa en el prurito en comparación con el placebo. La parte confirmatoria del estudio está en curso, con la inscripción prevista para completarse en 2026.
La Designación de Terapia Innovadora tiene como objetivo acelerar el desarrollo y la revisión de medicamentos para enfermedades graves, requiriendo evidencia clínica preliminar de una mejora sustancial sobre las terapias existentes. La directora médica de Mirum, Dr. Joanne Quan, enfatizó la importancia de abordar el picor relacionado con la PBC y el compromiso de la empresa de hacer volixibat disponible para los pacientes lo más rápido posible.
Mirum Pharmaceuticals (Nasdaq: MIRM)은 FDA가 volixibat에게 1차 담즙성 콜라이틱 (PBC) 환자의 담즙성 가려움증 치료를 위한 혁신 치료제 지정을 부여했다고 발표했습니다. 이 지정은 위약에 비해 가려움증에서 통계적으로 유의미한 개선을 보인 2상 2b VANTAGE 연구의 긍정적인 중간 결과를 기반으로 합니다. 연구의 확인 부분은 진행 중이며, 등록은 2026년에 완료될 것으로 예상됩니다.
혁신 치료제 지정은 심각한 질병에 대한 약물의 개발 및 검토를 가속화하는 것을 목표로 하며, 기존 치료법보다 상당한 개선의 초기 임상 증거가 필요합니다. Mirum의 최고 의학 책임자인 Dr. Joanne Quan은 PBC 관련 가려움증 해결의 중요성과 환자에게 가능한 한 빨리 volixibat을 제공할 것에 대한 회사의 의지를 강조했습니다.
Mirum Pharmaceuticals (Nasdaq: MIRM) a annoncé que la FDA a accordé la désignation de thérapie novatrice à volixibat pour le traitement du prurit cholestatique chez les patients atteints de cholangite biliaire primitive (PBC). Cette désignation est basée sur des résultats intermédiaires positifs de l'étude de Phase 2b VANTAGE, qui a montré une amélioration statistiquement significative du prurit par rapport au placebo. La partie confirmatoire de l'étude est en cours, avec une inscription prévue jusqu'en 2026.
La désignation de thérapie novatrice vise à accélérer le développement et l'examen des médicaments pour les maladies graves, nécessitant des preuves cliniques préliminaires d'une amélioration substantielle par rapport aux thérapies existantes. La directrice médicale de Mirum, Dr. Joanne Quan, a souligné l'importance de traiter les démangeaisons liées à la PBC et l'engagement de l'entreprise à rendre volixibat disponible pour les patients le plus rapidement possible.
Mirum Pharmaceuticals (Nasdaq: MIRM) gab bekannt, dass die FDA die Durchbruch-Therapie-Bezeichnung für volixibat zur Behandlung des cholestatischen Juckreizes bei Patienten mit primärer biliärer Cholangitis (PBC) vergeben hat. Diese Bezeichnung basiert auf positiven Zwischenresultaten der Phase 2b VANTAGE-Studie, die eine statistisch signifikante Verbesserung des Juckreizes im Vergleich zu einem Placebo zeigte. Der Bestätigungsteil der Studie ist derzeit im Gange, mit einer erwarteten Registrierung bis 2026.
Die Durchbruch-Therapie-Bezeichnung zielt darauf ab, die Entwicklung und Überprüfung von Arzneimitteln für schwerwiegende Erkrankungen zu beschleunigen und erfordert vorläufige klinische Nachweise für eine wesentliche Verbesserung gegenüber bestehenden Therapien. Die leitende Ärztin von Mirum, Dr. Joanne Quan, betonte die Bedeutung der Bekämpfung des mit PBC verbundenen Juckreizes und das Engagement des Unternehmens, volixibat so schnell wie möglich für die Patienten verfügbar zu machen.
- FDA granted Breakthrough Therapy Designation for volixibat
- Positive interim results from Phase 2b VANTAGE study
- Statistically significant improvement in pruritus vs placebo
- Potential expedited development and review process
- Confirmatory portion of the study still ongoing
- Study enrollment not expected to complete until 2026
Insights
The FDA's Breakthrough Therapy Designation for volixibat is a significant milestone for Mirum Pharmaceuticals. This designation, based on positive interim Phase 2b results, could accelerate the drug's path to market for treating cholestatic pruritus in primary biliary cholangitis (PBC) patients. Key points:
- Volixibat showed statistically significant improvement vs. placebo in pruritus, addressing a major symptom of PBC
- The ongoing VANTAGE study is expected to complete enrollment by 2026, indicating a potentially lengthy development timeline
- This designation may lead to expedited review and development, potentially bringing the treatment to market faster
For investors, this news signals increased potential for volixibat to become a valuable asset in Mirum's portfolio. However, it's important to note that full trial results and regulatory approval are still pending and the timeline to market remains uncertain.
This Breakthrough Therapy Designation for volixibat could significantly impact Mirum Pharmaceuticals' market position and financial outlook:
- Potential for accelerated approval process, reducing time and costs to market
- Increased likelihood of successful drug development, potentially de-risking the investment in volixibat
- Enhanced competitive advantage in the PBC treatment market
- Possible increase in partnership or licensing opportunities
With a market cap of
- Designation based on positive interim analysis of Phase 2b VANTAGE study
“Breakthrough Therapy Designation for volixibat in PBC underscores the importance and urgency for a treatment to address one of the most burdensome impacts of this rare liver disease,” said Joanne Quan, MD, chief medical officer at Mirum. “We look forward to advancing our VANTAGE study with the goal of making volixibat available to patients living with PBC-related itch as quickly as possible.”
The FDA’s Breakthrough Therapy Designation aims to expedite the development and review of drugs that are intended to treat serious or life-threatening diseases. To qualify for this designation, preliminary clinical evidence must indicate that the drug may demonstrate substantial improvement over existing therapy on at least one clinically significant endpoint.
About Volixibat
Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study), and primary biliary cholangitis (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate.
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 CHENODAL® (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 debilitating liver 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. Lastly, chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the
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 results, conduct, progress and timing of Mirum’s clinical trials and the review and approval process by regulatory authorities of its product candidates. 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,” “would,” “potential” 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 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 Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors,” as well as in its subsequent reports on Form 8-K, all of which are filed with the
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Media Contact:
Erin Murphy
media@mirumpharma.com
Investor Contact:
Andrew McKibben
ir@mirumphama.com
Source: Mirum Pharmaceuticals, Inc.
FAQ
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