TARRYTOWN, N.Y. and PARIS, Feb. 9, 2021 /PRNewswire/ — Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the U.S. Food and Drug Administration (FDA) approved the PD-1 inhibitor Libtayo (cemiplimab-rwlc) as the first immunotherapy treatment indicated for patients with advanced basal cell carcinoma (BCC) previously treated with a hedgehog pathway inhibitor (HHI) or for whom an HHI is not appropriate. Full approval was granted for patients with locally advanced BCC and accelerated approval was granted for patients with metastatic BCC.
“Today’s FDA approval of Libtayo will change the treatment paradigm for patients with advanced basal cell carcinoma,” said Karl Lewis, M.D., Professor in the Division of Medical Oncology at the University of Colorado and a trial investigator. “Advanced basal cell carcinoma is a persistent, painful and highly disfiguring cancer. While the primary systemic treatment options are hedgehog inhibitors, many patients will eventually progress on or become intolerant to this therapy. With Libtayo, these patients now have a new immunotherapy option that has demonstrated clinically meaningful and durable anti-tumor responses.”
Libtayo is the first treatment to show a clinical benefit in patients with advanced BCC after HHI therapy in a pivotal trial. The full approval in locally advanced BCC is based on the primary analysis from the trial, and the accelerated approval in metastatic BCC is based on an interim analysis showing the impact of Libtayo on tumor response rate and durability of response. Continued approval in metastatic BCC may be contingent on additional data from the trial verifying clinical benefit.
“With today’s approval, Libtayo is now approved for both advanced cutaneous squamous cell and basal cell carcinomas, building a strong foundation in dermato-oncology,” said Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Oncology, at Regeneron. “Beyond skin cancers, we also continue to investigate the potential of Libtayo in other difficult-to-treat cancers, starting with non-small cell lung cancer where an FDA decision is expected by the end of February.”
This marks the second U.S. approval for Libtayo, and is based on FDA Priority Review, which is reserved for medicines that, if approved, would represent significant improvements in safety or efficacy in treating serious conditions. In 2018, Libtayo was approved as the first systemic treatment for adults with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation. Immune-mediated adverse reactions, which may be severe or fatal, can occur in any organ system or tissue during or after treatment with Libtayo.
BCC is the most common type of skin cancer in the U.S., with approximately two million new cases diagnosed every year. While the vast majority of BCCs are caught early and cured with surgery and radiation, a small proportion of tumors can become advanced and penetrate deep into surrounding tissues (locally advanced) or spread to other parts of the body (metastatic), which is more difficult to treat.
“Patients with advanced forms of basal cell carcinoma face a very difficult prognosis,” said Peter Adamson, Global Development Head, Oncology and Pediatric Innovation at Sanofi. “Thanks to the participation and support of researchers, clinicians, and patients around the world, we are proud to bring forward a new immunotherapy treatment option for appropriate patients in the U.S. affected by advanced BCC, another devastating non-melanoma skin cancer. Together with Regeneron, we continue to develop Libtayo in numerous clinical trials and settings, including as monotherapy and in combination with several other therapeutic approaches as part of our commitment to innovation towards meaningful treatment options for patients with significant unmet needs.”
Pivotal Clinical Trial Supporting the Approval
The FDA approval of Libtayo was based on an open-label, multi-center, non-randomized Phase 2 trial of patients with unresectable locally advanced BCC or metastatic BCC (nodal or distant). This was the largest prospective clinical trial (n=132) among this patient population, with 112 patients included in the efficacy analysis. Patients in both cohorts had either progressed on HHI therapy, had not had an objective response after 9 months on HHI therapy, or were intolerant of prior HHI therapy.
The primary efficacy endpoint was confirmed objective response rate (ORR) and a key secondary endpoint was duration of response (DOR), assessed by independent central review.
Efficacy results for patients treated with Libtayo 350 mg every 3 weeks were clinically meaningful and durable, with specific data as follows:
(95% confidence interval [CI])
Complete response (CR)
Partial response (PR)
Median DOR in months (range)
Patients with observed DOR > 6 months, n (%)
+ Denotes ongoing at last assessment
a Median duration of follow up: mBCC 9.5 months; laBCC 15.1 months
b With longer follow-up, ORR in laBCC increased to 26 patients (31%) as reported at ESMO 2020
Among patients evaluable for safety (n=132), the most common adverse reactions reported in at least 15% of patients were fatigue (49%), musculoskeletal pain (33%), diarrhea (25%), rash (22%), pruritus (20%) and upper respiratory tract infection (15%). Serious adverse reactions occurred in 32% of patients; those occurring in at least two patients included urinary tract infection, colitis, acute kidney injury, adrenal insufficiency, anemia, infected neoplasm and somnolence. Adverse reactions resulting in permanent discontinuation occurred in 13% of patients, with the most common reactions (occurring in at least two patients) being colitis and general physical health deterioration.
Libtayo is a fully-human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T-cells. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation.
The recommended dose of Libtayo is 350 mg administered as an intravenous infusion over 30 minutes every three weeks, until disease progression or unacceptable toxicity. Libtayo is available as a single-dose 350 mg vial. No PD-L1 or tumor mutational burden (TMB) testing is required before starting treatment with Libtayo for advanced BCC.
In the U.S., the generic name for Libtayo in its approved indications is cemiplimab-rwlc, with rwlc as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the FDA. Outside of the U.S., the generic name for Libtayo in its approved indication is cemiplimab.
Libtayo was invented using Regeneron’s VelocImmune® technology that utilizes a proprietary genetically-engineered mouse platform endowed with a genetically-humanized immune system to produce optimized fully-human antibodies. VelocImmune technology has also been used to create multiple antibodies including Dupixent® (dupilumab), Praluent® (alirocumab), Kevzara® (sarilumab), Inmazeb™ (atoltivimab, maftivimab, and odesivimab-ebgn) and Regeneron’s antibody cocktail for COVID-19, which was recently granted Emergency Use Authorization (EUA) in the U.S.
About the Libtayo Development Program
Libtayo is currently under Priority Review by the FDA for advanced non-small cell lung cancer with ≥50% PD-L1 expression, with a target action date of February 28, 2021. The European Medicines Agency (EMA) is assessing Libtayo in both advanced NSCLC with ≥50% PD-L1 expression and locally advanced BCC following treatment with a hedgehog inhibitor, and decisions from the European Commission are expected in mid-2021.
The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. In skin cancer, this includes trials in adjuvant and neoadjuvant CSCC. Libtayo is also being investigated in pivotal trials in NSCLC (in combination with chemotherapy) and cervical cancer, as well as in trials combining Libtayo with either conventional or novel therapeutic approaches for both solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.
Libtayo is being jointly developed by Regeneron and Sanofi under a global collaboration agreement.
What is Libtayo?
Libtayo is a prescription medicine used to treat people with a type of skin cancer called cutaneous squamous cell carcinoma (CSCC) that has spread or cannot be cured by surgery or radiation.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that cannot be removed by surgery (locally advanced BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with a HHI.
Libtayo is a prescription medicine used to treat people with a type of skin cancer called basal cell carcinoma that has spread (metastatic BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with a HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.
About Regeneron Pharmaceuticals, Inc.
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents life-transforming medicines for people with serious diseases. Founded and led for over 30 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to eight FDA-approved treatments and numerous product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune which uses unique genetically-humanized mice to produce optimized fully-human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.
For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter.
Sanofi is dedicated to supporting people through their health challenges. We are a global biopharmaceutical company focused on human health. We prevent illness with vaccines, provide innovative treatments to fight pain and ease suffering. We stand by the few who suffer from rare diseases and the millions with long-term chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is transforming scientific innovation into healthcare solutions around the globe.
Sanofi, Empowering Life
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These statements concern, and these risks and uncertainties include, among others, the impact of SARS-CoV-2 (the virus that has caused the COVID-19 pandemic) on Regeneron’s business and its employees, collaborators, and suppliers and other third parties on which Regeneron relies, Regeneron’s and its collaborators’ ability to continue to conduct research and clinical programs, Regeneron’s ability to manage its supply chain, net product sales of products marketed or otherwise commercialized by Regeneron and/or its collaborators (collectively, “Regeneron’s Products”), and the global economy; the nature, timing, and possible success and therapeutic applications of Regeneron’s Products and product candidates being developed by Regeneron and/or its collaborators (collectively, “Regeneron’s Product Candidates”) and research and clinical programs now underway or planned, including without limitation Libtayo® (cemiplimab) for the treatment of locally advanced or metastatic basal cell carcinoma; uncertainty of market acceptance and commercial success of Regeneron’s Products and Regeneron’s Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary), including the studies discussed or referenced in this press release, on the commercial success of Regeneron’s Products (such as Libtayo) and Regeneron’s Product Candidates; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron’s Product Candidates and new indications for Regeneron’s Products, such as Libtayo for the treatment of locally advanced or metastatic non-small cell lung cancer, adjuvant and neoadjuvant cutaneous squamous cell carcinoma, and cervical cancer (as well as in combination with either conventional or novel therapeutic approaches for both solid tumors and blood cancers); safety issues resulting from the administration of Regeneron’s Products (such as Libtayo) and Regeneron’s Product Candidates in patients, including serious complications or side effects in connection with the use of Regeneron’s Products and Regeneron’s Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron’s ability to continue to develop or commercialize Regeneron’s Products and Regeneron’s Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron’s Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement of Regeneron’s Products from third-party payers, including private payer healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payers and new policies and procedures adopted by such payers; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron’s Products and Regeneron’s Product Candidates; the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; the ability of Regeneron to manufacture and manage supply chains for multiple products and product candidates; the ability of Regeneron’s collaborators, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron’s Products and Regeneron’s Product Candidates; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron’s agreements with Sanofi, Bayer, and Teva Pharmaceutical Industries Ltd. (or their respective affiliated companies, as applicable), to be cancelled or terminated; and risks associated with intellectual property of other parties and pending or future litigation relating thereto (including without limitation the patent litigation and other related proceedings relating to EYLEA® (aflibercept) Injection, Dupixent® (dupilumab), Praluent® (alirocumab), and REGEN-COVTM (casirivimab and imdevimab)), other litigation and other proceedings and government investigations relating to the Company and/or its operations, the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron’s business, prospects, operating results, and financial condition. A more complete description of these and other material risks can be found in Regeneron’s filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2020. Any forward-looking statements are made based on management’s current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise.
Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron’s media and investor relations website (http://newsroom.regeneron.com) and its Twitter feed (http://twitter.com/regeneron).
Sanofi Forward-Looking Statements
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SOURCE Regeneron Pharmaceuticals, Inc.
Posted: February 2021
- FDA Approves Libtayo (cemiplimab-rwlc) for Advanced Cutaneous Squamous Cell Carcinoma – September 28, 2018
- FDA to Conduct Priority Review of Cemiplimab as a Potential Treatment for Advanced Cutaneous Squamous Cell Carcinoma – April 30, 2018
Libtayo (cemiplimab-rwlc) FDA Approval History
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