FDA Approvals: Therapies for Lupus, MS, Cancer and a Genomic Alteration in Pigs

FDA Approval

The U.S. Food and Drug Administration (FDA) had several approvals this week. Read on to see what the regulatory agency gave the go-ahead to.


GSK announced on Thursday that the U.S. Food and Drug Administration (FDA) has approved belimumab (Benlysta®), the first-ever treatment for adults with lupus nephritis (LN) who are currently receiving standard therapy.

LN, or inflammation of the kidneys, is a common system of systemic lupus erythematosus (SLE), one of the most prevalent autoimmune diseases. Lupus causes the production of proteins called autoantibodies in the immune system, which attack tissues and organs, including the kidneys. Five out of 10 adults living with lupus will have LN, while eight out of 10 children with lupus are found to have kidney damage, usually caused by LN.

The Alliance for Lupus Research (LRA) President and Chief Executive Officer Kenneth M. Farber extended congratulations to GSK on behalf of the alliance:

"An effective treatment developed specifically for lupus nephritis has been desperately needed. We are particularly proud to see this approval for another indication of belimumab, having funded much of the original research that led to the drug's development. We at the LRA are thrilled to share this news about belimumab and look forward to future approvals of other lupus nephritis treatment options.”

Approval was given following positive results of a 104-week BLISS-LN phase III post-approval study which showed belimumab to be a safe and effective therapy for treating LN.

"Approximately 40% of patients with systemic lupus erythematosus develop lupus nephritis, which causes inflammation in the kidneys and can lead to end-stage kidney disease.  BENLYSTA is the first medicine approved to treat systemic lupus and adults with active lupus nephritis, an important treatment advance for patients with this incurable autoimmune disease,” said GSK Chief Scientific Officer and President of R&D Dr. Hal Barron.

First approved in 2011, belimumab is now the first and only biologic approved for both SLE and LN in over a half-century.

MacroGenics Inc.

MacroGenics, a biopharma company working to make cancer history with the development of innovative monoclonal antibody-based therapeutics, had its first product, MARGENZA™, approved by the FDA on Wednesday.

MARGENZA (margetuximab-cmkb), was given the green light, in combination with chemotherapy, the treatment of adult patients with metastatic HER2-positive breast cancer.

An Fc-engineered monoclonal antibody targeting the HER2 oncoprotein, MARGENZA offers hope for patients who have received two or more prior anti-HER2 regimens, with at least one being for metastatic disease.

In a pivotal head-to-head Phase III SOPHIA clinical trial, it became the first HER2-targeted therapy to improve progression-free survival (PFS) versus Herceptin® (trastuzumab), when each was combined with chemotherapy. In comparison with trastuzumab plus chemotherapy, MARGENZA showed a statistically significant 24% reduction in the risk of disease progression or death, with an objective response rate of 22% compared with 16% for trastuzumab.

“The approval of MARGENZA is an exciting milestone for MacroGenics and, more importantly, it brings a new treatment option to metastatic breast cancer patients. We are grateful for the patients who participated in this study, as well as their families, and everyone who played a role in helping MacroGenics reach this milestone,” said MacroGeneics president and CEO Scott Koenig, M.D., Ph.D., at the time of the announcement.

The product launch is expected to take place in March of 2021.

Athenex, Inc.

There will be a brand-new treatment for actinic keratosis on the market next year, with Tuesday’s FDA approval of Athenix’s Klisyri® (tirbanibulin).

This will be the first branded proprietary product for Athenex, which will be launched in the U.S. in partnership with Barcelona-based biopharma company, Almirall. Klisyri will be manufactured by Athenex.

Actinic Keratosis, a rough, scaly skin patch occurring after years of sun exposure, has the potential to turn into squamous cell skin cancer.

The approval is based on two pivotal, randomized, double-blind, vehicle-controlled Phase III studies (KX01-AK-003 and KX01-AK-004) in 702 adults with actinic keratosis of the face or scalp where tirbanibulin demonstrated complete clearance of lesions at day 57 in treated areas in a significantly higher number of patients compared to vehicle.

“The FDA approval of Klisyri is a significant milestone for Athenex. Klisyri is a home-grown product discovered and characterized by Athenex scientists and developed from pre-IND to NDA by the Athenex team. We are extremely proud of our team’s excellent execution. Approval demonstrates our ability to execute upon the entirety of the drug development and registration process. We are excited to partner with Almirall to bring this first-in-class microtubule inhibitor to patients with actinic keratosis in the U.S.,” said Athenex Chairman and Chief Executive Officer, Dr. Johnson Lau.

Buffalo, New York-based Athenex is dedicated to the discovery, development and commercialization of novel therapies for the treatment of cancer.

Genentech Inc. (Roche)

Genentech (a member of the Roche group) began the week with a bang, as the FDA on Monday approved a shorter, two-hour infusion time for relapsing and primary progressive multiple sclerosis therapy Ocrevus® (ocrelizumab). 

Ocrelizumab is a humanized monoclonal antibody designed to target CD20-positive B cells, an immune cell thought to be a key cause of the myelin and axonal damage found in multiple sclerosis. It is the first and only therapy approved for both relapsing MS (RMS), relapsing-remitting MS (RRMS), secondary-progressive MS (SPMS), and primary-progressive MS (PPMS). 

“More than 170,000 people with MS have been treated with Ocrevus – the only approved B-cell therapy with a twice-yearly dosing schedule – and it is the most prescribed MS medicine in the U.S., We constantly strive to improve the experience that patients and their physicians have with our medicines, and we believe people with relapsing and primary progressive MS will find the shorter two-hour Ocrevus infusion time to be more convenient,” said Genentech Chief Medical Officer and Head of Global Product Development, Levi Garraway, M.D., Ph.D.

Approval was given on the strength of a randomized, double-blind ENSEMBLE PLUS study, which met the primary endpoint, showing similar frequency and severity of IRs for a two-hour Ocrevus infusion time vs. the already approved 3.5-hour time in patients with RRMS. Importantly, a consistent safety profile was maintained.

The new two-hour infusion time was previously approved by the European Medicines Agency (EMA) in May 2020.

When Pigs Fly Allergy-friendly

The week began with big news from the field of genomics, as the FDA on Monday approved a first-of-its-kind intentional genomic alteration (IGA) in pigs that could reduce potential allergic reactions when porcine materials are used for medical purposes in humans.

The alteration was pulled off in a line of domestic pigs called GalSafe pigs, which may be used for either food or human therapeutics.

"Today's first-ever approval of an animal biotechnology product for both food and as a potential source for biomedical use represents a tremendous milestone for scientific innovation," FDA Commissioner Stephen M. Hahn, M.D. said in a statement.

The IGA is meant to eliminate alpha-gal sugar on the surface of the pigs’ cells, thereby making the resulting food or therapeutics safe for use in people with Alpha-gal syndrome (AGS) which can cause a mild or severe allergic reaction to alpha-gal sugar found in red meat.

The FDA reviewed data that demonstrated that there is no detectable level of alpha-gal sugar across multiple generations of GalSafe pigs.

This particular pig, however, has not been evaluated for use in xenotransplantation products or for transplantation or implantation into humans.

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