Orphan Drug Designation Granted to Rezafungin in EU for the Treatment of Invasive Candidiasis
Mundipharma and Cidara Therapeutics today announced that the European Commission (EC) has now adopted the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) recommendation to grant Orphan Drug Designation (ODD) to rezafungin, a novel, once-weekly echinocandin, for the treatment of invasive candidiasis (IC).4
The EMA considers ODD status for medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than five per 10,000 people in the EU.5 It is estimated that IC occurs in around 0.84 per 10,000 people in the EU,6 and mostly affects hospitalised, immunocompromised or critically ill patients.2
IC continues to be an area of significant unmet need, especially for critically ill patients in hospitals and patients with compromised immune systems. Despite a number of available treatments, mortality rates are as high as 40%.3 IC is characterised as a severe, life-threatening systemic Candida infection of the bloodstream and/or deep/visceral tissues, known as candidaemia and deep-seated tissue candidiasis.1
Brian Sheehan, Ph.D., Chief Scientific Officer, Mundipharma, commented: “Orphan drug designation is an important milestone in the development of rezafungin, which is currently in phase 3 clinical trials.Fungal infections still pose a major threat to the lives of hospitalised or immunocompromised patients. We are proud that patients affected with invasive candidiasis may have an additional treatment option to treat this potentially life-threatening condition.”
The EC decision adopting the COMP opinion follows that of the U.S. Food and Drug Administration (FDA), which has already designated rezafungin as a Qualified Infectious Disease Product (QIDP) with Fast Track status and ODD for its use in the treatment of IC, including candidaemia.
Jeffrey Stein, Ph.D., President and Chief Executive Officer, Cidara, added: “We are pleased by the decision of the EMA to grant orphan drug designation to rezafungin, further supporting its potential as the first new antifungal for the treatment of serious invasive Candida infections in nearly 15 years. We look forward to continuing to work closely with our colleagues at Mundipharma to advance rezafungin through late-stage clinical development.”
To find out more about the rezafungin clinical trials programme, please visit: https://clinicaltrials.gov/ct2/results?cond=&term=rezafungin&cntry=&state=&city=&dist=&Search=Search
Rezafungin is a novel once-weekly echinocandin being developed for both the treatment and prevention of severe fungal infections. Echinocandins are a class of antifungal drugs that act by inhibiting β (1, 3)-D- glucan synthase, a key enzyme necessary for integrity of the fungal cell wall.7
In studies to date, rezafungin has demonstrated a unique pharmacokinetic profile with a prolonged half-life and is also dosed for front-loaded plasma exposure which, in contrast to all other echinocandins, is intended to allow for once-weekly IV therapy for inpatient and outpatient use.8In Vitro data demonstrate that rezafungin has potent antifungal activity against representative strains of Candida spp, Aspergillus spp, and Pneumocystis spp.9,10 Data from the Phase 2 STRIVE study showed that rezafungin met all of its objectives for safety, tolerability and efficacy in the treatment of patients with candidaemia and/or IC.11
Rezafungin is currently in Phase 3 development. The ReSTORE trial (NCT03667690) is an ongoing, global, randomised, double-blind, controlled, pivotal Phase 3 study to evaluate the safety, tolerability and efficacy of rezafungin compared to caspofungin for the treatment of candidemia and IC in approximately 184 qualifying patients. The primary efficacy outcome measure for the EMA is Global Cure at Day 14 while the primary efficacy outcome measure for the FDA is All-Cause Mortality at Day 30. The ReSPECT trial (NCT04368559) is an ongoing global, randomised, double-blind, controlled, pivotal Phase 3 trial of rezafungin versus the standard antimicrobial regimen to prevent invasive fungal disease due to Candida, Aspergillus and Pneumocystis in subjects undergoing allogeneic blood and marrow transplantation. The trial is expected to enroll approximately 462 adults with underlying conditions, and receive either rezafungin or the standard antimicrobial regimen for 90 days, at which time fungal-free survival will be measured as the primary efficacy outcome.
Mundipharma is a global (ex-US) network of independent associated companies with a presence across Africa, Asia Pacific, Canada, Europe, Latin America and the Middle East.
As a dynamic, forward-looking organisation we are dedicated to bringing innovative treatments to patients in the areas of Pain & Supportive Care and Consumer Healthcare as well as other severe and debilitating disease areas.
Our guiding principles, centred around Integrity and Patient-Centricity, are at the heart of everything we do. For more information visit www.mundipharma.com
About Cidara Therapeutics
Cidara is developing long-acting therapeutics designed to transform the standard of care for patients facing serious fungal or viral infections. The Company’s portfolio is comprised of its lead antifungal candidate, rezafungin, in addition to antiviral conjugates (AVCs) for the prevention and treatment of influenza and other viral diseases from Cidara’s proprietary Cloudbreak® antiviral platform. Cidara is headquartered in San Diego, California. For more information, please visit www.cidara.com.
This release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. “Forward-looking statements” describe future expectations, plans, results, or strategies and are generally preceded by words such as “anticipates,” “expect,” “may,” “plan” or “will”. Forward-looking statements in this release include, but are not limited to, statements related to rezafungin’s efficacy and potential as a once-weekly treatment and its ability to prevent severe fungal infections and disease. Such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements. These and other risks are identified under the caption “Risk Factors” in Cidara’s most recent Quarterly Report on Form 10-Q and other filings subsequently made 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. Cidara does not undertake any obligation to publicly update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise.
- Cortes JA, Corrales IF. Invasive Candidiasis: Epidemiology and Risk Factors. November 2018. Available at https://www.intechopen.com/books/fungal-infection/invasive-candidiasis-epidemiology-and-risk-factors. Last accessed December 2020.
- Arendrup MC, Patterson TF. Multidrug-Resistant Candida: Epidemiology, Molecular Mechanisms, and Treatment. J Infect Dis 2017;216(3):S445-S451. Available at https://pubmed.ncbi.nlm.nih.gov/28911043/. Last accessed December 2020.
- Kullberg BJ, Arendrup MC. Invasive Candidiasis. N Engl J Med 2015; 373:1445-1456.
- European Commission. Community Register of orphan medicinal products. Available at: https://ec.europa.eu/health/documents/community-register/html/o2385.htm Last accessed 21 January 2021.
- European Medicines Agency. Applying for orphan designation. Available at https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview. Last accessed December 2020.
- Data on file – Mundipharma application for orphan medicinal product designation, October 2020.
- Drugs.com. Echinocandins: What are Echinocandins? Available at https://www.drugs.com/drug-class/echinocandins.html. Last accessed December 2020.
- Zhao Y, Perlin DS. Review of the Novel Echinocandin Antifungal Rezafungin: Animal Studies and Clinical Data J. Fungi 2020;6:192.
- Pfaller MA, Carvalhaes C, Messer SA, et al. Activity of a long-acting echinocandin, rezafungin, and comparator antifungal agents tested against contemporary invasive fungal isolates (SENTRY Program, 2016 to 2018). Antimicrob Agents Chemother 2020 64:e00099-20.
- Ong V, Ashbaugh A, Cushion MT. Rezafungin Prevention of Pneumocystis Pneumonia and Pneumocystis Reactivation Using Different Doses and Durations of Prophylaxis in a Mouse Model. Biology of Blood and Marrow Transplantation 2020;26(3):S328-S329.
- Thompson GR, Soriano A, Skoutelis A, et al. Rezafungin versus Caspofungin in a Phase 2, Randomized, Double-Blind Study for the Treatment of Candidemia and Invasive Candidiasis- The STRIVE Trial. Clinical Infectious Diseases, ciaa1380, https://doi.org/10.1093/cid/ciaa1380.
Corporate Communications, Mundipharma
+44 (0)777 300 9578
Tel: +44 (0) 23 81 247 327
Karen O’Shea, Ph.D.
About Business Wire
Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Moody’s Analytics Wins IFRS 9 Solution Provider of the Year5.3.2021 08:30:00 CET | Press release
Moody’s Analytics has won IFRS 9 Solution Provider of the Year in the 2021 Insurance Asset Risk Awards. It is the latest recognition for our capabilities in this space following wins for IFRS 9 – Enterprise Solution of the Year and IFRS 9 – ECL Modelling Solution of the Year at last year’s Risk Technology Awards. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210304006204/en/ Central to our offering for insurers is the ImpairmentStudio™ for IFRS 9 solution, a cloud-based platform that brings together our award-winning economic scenarios, data, models, and enterprise software. Financial institutions use it to automate the credit loss impairment calculations mandated by the new IFRS 9 accounting standard. For insurers, these new financial reporting requirements call for much more granular data than was previously needed. The Moody’s Analytics offering helps them access and use all of the required data while managing and monito
C3.ai Digital Transformation Institute Announces Shell as New Industry Partner4.3.2021 20:00:00 CET | Press release
The C3.ai Digital Transformation Institute (DTI) today announced that global energy company Shell is the newest industry partner to join the consortium of universities, national laboratories, and companies that make up the Institute. Shell’s Chief Scientist - Computation and Data Science Detlef Hohl will represent the company in the consortium. The DTI Industry Partner Program enables leading companies from around the world to engage with DTI researchers and activities and to contribute data sets that will be available for research after anonymization. Industry partners also are encouraged to engage with Institute researchers and collaborate on research projects as well as participate in DTI conferences. DTI recently released its second call for proposals to fund advanced research projects focused on applying digital transformation and AI to address energy and climate security. “Shell has established clear leadership in new energy, having laid out its ‘Powering Progress’ commitment to
Dole Launches “The Dole Way” Sustainability Campaign4.3.2021 19:22:00 CET | Press release
Dole Food Company announced today the launching of an integrated sustainability campaign aimed at amplifying its commitment to the key enterprise-wide sustainability goals launched in 2020 under “The Dole Way.” As a leader in the produce industry for over 150 years, Dole believes in acting on its promise to increase sustainability, transparency, corporate responsibility and trust. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210304005912/en/ The Dole Way framework paves the way for further improvements in areas where Dole believes it can make the biggest positive impacts (Photo: Business Wire) “The Dole Way” campaign kicked-off with a live, internal event on February 25, led by Dole’s president and chief executive officer, Johan Linden, and will be sustained over the next several months through integrated earned and owned social media efforts. “The Dole Way” campaign illustrates a path of transformation that will recognize
Milliken & Company Appoints Cindy Boiter as Executive Vice President, Chemical Division President4.3.2021 18:00:00 CET | Press release
Milliken & Company is pleased to announce Cindy Boiter has been promoted to executive vice president and president of Milliken’s Chemical Division. Effective March 15, she will step into the role currently held by David Moody as he transitions to a role outside of Milliken. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210304005823/en/ Cindy Boiter, newly announced executive vice president and president of Milliken's Chemical Division (Photo: Business Wire) “It is an exciting appointment for our company, as Cindy will become the first-ever woman division president at Milliken,” shares Halsey Cook, president and CEO for Milliken. “She is uniquely qualified to lead our chemical division thanks to her extensive experience in the division, the acquisitions she has completed and her embodiment of Milliken’s purpose.” Boiter joined Milliken’s Chemical Division in 2012 as marketing, strategy and business development director and p
Verimatrix Takes Home Two 2021 Gold Cybersecurity Excellence Awards4.3.2021 17:45:00 CET | Press release
Regulatory News: Verimatrix, (Paris:VMX) (Euronext Paris: VMX), the leader in powering the modern connected world with people-centered security, today announced it won two gold awards in this year’s Cybersecurity Excellence Awards program. Verimatrix Multi-DRM won in the Digital Rights Management (North America) category while Verimatrix’s Application Shielding suite of products were recognized in the Automotive Security IoT (North America) category. It marks the third straight year the company has received honors from the award program that pays tribute to a wide variety of cybersecurity companies spanning the world’s largest vendors to the most recent startups. Verimatrix’s code shielding and embedded security capabilities picked up kudos in previous years. “The automotive industry stands as a superb example of a vertical where Verimatrix uniquely addresses multiple cybersecurity needs – in this case, vital components such as keyless entry mobile apps and infotainment systems, said A
Andersen Global Strengthens Presence with Jordanian Tax Firm4.3.2021 15:30:00 CET | Press release
Andersen Global enters into a Collaboration Agreement with Amman-based tax firm Al-Tillawi & Al-Khateeb Co., adding dimension to the existing capabilities of the organization’s member firm in Jordan. Founded in 1996, Al-Tillawi & Al-Khateeb Co. provides a wide range of tax and advisory services including corporate and family business, employment, mergers and acquisitions, outsourcing and social security as well as IT solutions. The firm, led by Managing Partner Waleed Al Tillawi, serves a broad clientele in industries such as power and energy, technology and communication, hotel and tourism, finance and retail. “Our team’s dedication to transparency and independence have been the driving forces behind the high-quality client services we provide,” Waleed said. “Collaborating with like-minded individuals at Andersen Global will allow us to take our service offerings to the next level and expand our reach more broadly. We look forward to working with the organization’s member and collabor
Business Finland Awarded Funding for Medicortex4.3.2021 15:08:00 CET | Press release
Medicortex Finland Oy, a Finnish biotechnology company focusing on brain injury diagnostics and drug development, announces receiving funding from the Finnish government innovation funding branch Business Finland. The funds are meant to expedite the development of a medical diagnostic test to improve detection of traumatic brain injury (TBI) and concussion. Medicortex has discovered novel biomarkers of head injury in body fluids such as saliva and urine. The funding which covers 50% of the expenses will support a project consisting of the development of a test kit for easy and rapid detection of brain injury and concussion. The objective of the company’s biomarker program is to generate new means for early detection of brain injury, which is a global unresolved issue. Current methods of brain injury diagnostics which are based on neurological examination and imaging do not reliably detect mild injury, yet they can have implications that are fatal or develop into a chronic condition if
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.Visit our pressroom