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Regeneron's Applications for gMG Drug Accepted by FDA and EMA
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Key Takeaways
Regeneron's cemdisiran NDA for gMG received FDA Priority Review with a November 2026 action date.
Phase III NIMBLE met primary and key secondary endpoints with every-12-week subcutaneous dosing.
REGN aims to diversify beyond Eylea as cemdisiran could expand its portfolio if approved.
Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that both the FDA and European Medicines Agency (“EMA”) have accepted the regulatory applications seeking approval of cemdisiran to treat adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.
The FDA will review the new drug application under Priority Review with a target action date in November 2026, following use of a Priority Review Voucher.
A decision from the European Commission is anticipated in the second half of 2027.
Upon gaining potential approval, cemdisiran could be the first siRNA approved for the treatment of gMG and only therapy to be offered subcutaneously with four times a year dosing.
Regeneron’s shares have lost 20.7% so far this year compared with the industry’s decline of 0.2%.
Image Source: Zacks Investment Research
More on REGN’s Submissions for Cemdisiran
Regeneron’s submissions are supported by positive data from the phase III NIMBLE study.
This late-stage study was evaluating cemdisiran, dosed subcutaneously every 12 weeks, in adults with symptomatic gMG who may be receiving standard-of-care immunosuppressants based on the investigator’s discretion.
The NIMBLE study met both its primary and key secondary endpoints at week 24, highlighting the potential of the therapy to deliver best-in-class efficacy along with a convenient treatment regimen for patients with gMG.
Myasthenia gravis (MG) is a rare, chronic autoimmune disorder, wherein abnormal anti-AChR antibodies activate the complement system, including C5, disrupting communication between nerves and muscles that results in debilitating and potentially life-threatening muscle weakness.
MG affects an estimated 150 to 200 individuals per million worldwide, including approximately 85,000 people in the United States. The disease often begins with ocular symptoms, but about 85% of patients experience progression to additional disease manifestations, which is then categorized as gMG.
Patients with gMG commonly experience extreme fatigue, as well as difficulties with facial movements, speech, swallowing and mobility.
Despite available treatment options, many patients continue to face significant challenges in disease management. Current therapies may primarily address symptoms rather than the underlying disease, while long-term use of immunosuppressive agents can be associated with substantial treatment burden. In addition, some patients experience inadequate responses or a loss of treatment effectiveness over time, further impacting quality of life.
A regulatory filing in Japan is also planned for early 2027.
Please note that Regeneron is solely responsible for the development, manufacturing, and commercialization of cemdisiran as a monotherapy and in combination with C5 antibodies through a worldwide licensing agreement with Alnylam (ALNY - Free Report) .
Regeneron and ALNY have collaborated to discover, develop, and commercialize RNAi therapeutics for numerous diseases by addressing therapeutic disease targets expressed in the eye and central nervous system, in addition to a select number of targets expressed in the liver.
REGN’s Efforts to Diversify Portfolio
Regeneron is actively working to diversify its revenue base and reduce dependence on lead drug Eylea.
Eylea is approved for various ophthalmology indications (neovascular age-related macular degeneration, diabetic macular edema and macular edema, among others). Regeneron co-developed Eylea with Bayer AG (BAYRY - Free Report) .
Eylea sales are under pressure amid intensifying competition from Roche’s Vabysmo, which has seen strong and rapid uptake. Vabysmo was designed to inhibit both Ang-2 and VEGF-A pathways, offering a differentiated mechanism that has resonated with physicians.
To counter the decline in Eylea sales, Regeneron developed a higher dose of the drug. The uptake of Eylea HD has been strong.
Regeneron records net product sales of Eylea and Eylea HD in the United States and Bayer does the same outside the country.
Apart from Eylea, profits from the sales of asthma drug Dupixent are a primary growth driver for REGN.
Regeneron has a collaboration agreement with Sanofi (SNY - Free Report) for drugs like Dupixent and Kevzara.
While Sanofi records sales, Regeneron registers its share of profits/losses in connection with the global sales of the aforementioned drugs.
The company’s oncology franchise is also gaining momentum driven by Libtayo.
Approval of additional drugs will broaden its diverse portfolio.
View the Zacks #1 Rank List
Image: Bigstock
Regeneron's Applications for gMG Drug Accepted by FDA and EMA
Key Takeaways
Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that both the FDA and European Medicines Agency (“EMA”) have accepted the regulatory applications seeking approval of cemdisiran to treat adult patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody-positive.
The FDA will review the new drug application under Priority Review with a target action date in November 2026, following use of a Priority Review Voucher.
A decision from the European Commission is anticipated in the second half of 2027.
Upon gaining potential approval, cemdisiran could be the first siRNA approved for the treatment of gMG and only therapy to be offered subcutaneously with four times a year dosing.
Regeneron’s shares have lost 20.7% so far this year compared with the industry’s decline of 0.2%.
Image Source: Zacks Investment Research
More on REGN’s Submissions for Cemdisiran
Regeneron’s submissions are supported by positive data from the phase III NIMBLE study.
This late-stage study was evaluating cemdisiran, dosed subcutaneously every 12 weeks, in adults with symptomatic gMG who may be receiving standard-of-care immunosuppressants based on the investigator’s discretion.
The NIMBLE study met both its primary and key secondary endpoints at week 24, highlighting the potential of the therapy to deliver best-in-class efficacy along with a convenient treatment regimen for patients with gMG.
Myasthenia gravis (MG) is a rare, chronic autoimmune disorder, wherein abnormal anti-AChR antibodies activate the complement system, including C5, disrupting communication between nerves and muscles that results in debilitating and potentially life-threatening muscle weakness.
MG affects an estimated 150 to 200 individuals per million worldwide, including approximately 85,000 people in the United States. The disease often begins with ocular symptoms, but about 85% of patients experience progression to additional disease manifestations, which is then categorized as gMG.
Patients with gMG commonly experience extreme fatigue, as well as difficulties with facial movements, speech, swallowing and mobility.
Despite available treatment options, many patients continue to face significant challenges in disease management. Current therapies may primarily address symptoms rather than the underlying disease, while long-term use of immunosuppressive agents can be associated with substantial treatment burden. In addition, some patients experience inadequate responses or a loss of treatment effectiveness over time, further impacting quality of life.
A regulatory filing in Japan is also planned for early 2027.
Please note that Regeneron is solely responsible for the development, manufacturing, and commercialization of cemdisiran as a monotherapy and in combination with C5 antibodies through a worldwide licensing agreement with Alnylam (ALNY - Free Report) .
Regeneron and ALNY have collaborated to discover, develop, and commercialize RNAi therapeutics for numerous diseases by addressing therapeutic disease targets expressed in the eye and central nervous system, in addition to a select number of targets expressed in the liver.
REGN’s Efforts to Diversify Portfolio
Regeneron is actively working to diversify its revenue base and reduce dependence on lead drug Eylea.
Eylea is approved for various ophthalmology indications (neovascular age-related macular degeneration, diabetic macular edema and macular edema, among others). Regeneron co-developed Eylea with Bayer AG (BAYRY - Free Report) .
Eylea sales are under pressure amid intensifying competition from Roche’s Vabysmo, which has seen strong and rapid uptake. Vabysmo was designed to inhibit both Ang-2 and VEGF-A pathways, offering a differentiated mechanism that has resonated with physicians.
To counter the decline in Eylea sales, Regeneron developed a higher dose of the drug. The uptake of Eylea HD has been strong.
Regeneron records net product sales of Eylea and Eylea HD in the United States and Bayer does the same outside the country.
Apart from Eylea, profits from the sales of asthma drug Dupixent are a primary growth driver for REGN.
Regeneron has a collaboration agreement with Sanofi (SNY - Free Report) for drugs like Dupixent and Kevzara.
While Sanofi records sales, Regeneron registers its share of profits/losses in connection with the global sales of the aforementioned drugs.
The company’s oncology franchise is also gaining momentum driven by Libtayo.
Approval of additional drugs will broaden its diverse portfolio.
REGN’s Zacks Rank
Regeneron currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.