We use cookies to understand how you use our site and to improve your experience.
This includes personalizing content and advertising.
By pressing "Accept All" or closing out of this banner, you consent to the use of all cookies and similar technologies and the sharing of information they collect with third parties.
You can reject marketing cookies by pressing "Deny Optional," but we still use essential, performance, and functional cookies.
In addition, whether you "Accept All," Deny Optional," click the X or otherwise continue to use the site, you accept our Privacy Policy and Terms of Service, revised from time to time.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Regeneron Secures FDA Approval for Rare Genetic Disorder Drug
Read MoreHide Full Article
Key Takeaways
Regeneron's Pasatru gained FDA approval to reduce new HO lesions and flare-ups in adults with FOP.
OPTIMA showed a 90% or greater reduction in new HO lesions at 56 weeks, with fewer flare-ups.
Regeneron plans a phase III pediatric study, while Pasatru faces EU review and potential global filings.
Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that the FDA has approved Pasatru (garetosmab-grts) to reduce the formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP).
FOP is an ultra-rare genetic disorder characterized by progressive formation of abnormal bone within muscles, tendons, ligaments and other connective tissues, a process known as HO.
Pasatru is a fully human monoclonal antibody derived using Regeneron’s VelocImmune technology. It works by blocking Activin A, a protein discovered by Regeneron scientists to play a critical role in the development of HO lesions in people with FOP.
Regeneron’s shares have gained 8.9% so far this year compared with the industry’s 7% growth.
Image Source: Zacks Investment Research
More on REGN’s Pasatru Approval
The approval was based on results from the OPTIMA study demonstrating a 90% or greater reduction in new HO lesions at 56 weeks with a dramatic reduction in clinician-assessed flare-ups in adults with FOP.
HO affecting the jaw, spine, hips and rib cage can make essential activities such as speaking, eating, walking and breathing increasingly difficult, ultimately resulting in severe and progressive loss of mobility.
FOP affects approximately 900 people worldwide, with most patients becoming wheelchair-dependent by age 30 and having a median survival of around 56 years.
Given the severe mobility challenges associated with FOP, Pasatru may be administered in various care settings, including at-home infusion when appropriate. The recommended starting dose is 10 mg/kg, administered intravenously more than 60 minutes once every four weeks. For patients who do not tolerate the starting dose, treatment may be reduced to three mg/kg on the same monthly schedule.
Meanwhile, a phase III study of Pasatru in adolescents and children with FOP, OPTIMA 2, is planned to begin later this year.
In the European Union (EU), Pasatru is currently under regulatory review by the European Medicines Agency (EMA), with additional submissions planned in other markets, including Japan. The drug has previously received FDA Fast Track and Orphan Drug designations, along with Orphan Designation from the EMA and Japan’s Ministry of Health, Labour and Welfare.
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, while Bayer records the same outside the country.
Apart from Eylea, profits from the sales of asthma drug Dupixent are a primary growth driver for REGN.
Regeneron Pharmaceuticals, Inc. Price, Consensus and EPS Surprise
Regeneron has a collaboration agreement with Sanofi (SNY - Free Report) for drugs like Dupixent and Kevzara.
While SNY 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 gaining momentum, driven by Libtayo.
Meanwhile, the FDA earlier granted accelerated approval to Otarmeni (lunsotogene parvec, formerly DB-OTO), the first gene therapy cleared under the FDA Commissioner’s National Priority Voucher Program. The adeno-associated virus-based treatment is indicated for pediatric and adult patients with severe-to-profound hearing loss caused by OTOF gene variants. A regulatory filing for the therapy is currently under review in the EU for the treatment of biallelic OTOF variant-associated hearing loss.
In June 2026, the FDA and EMA accepted Regeneron’s regulatory applications for cemdisiran, a C5-targeting RNAi therapy, for the treatment of adults with generalized myasthenia gravis. The FDA granted priority review to the filing, with a decision expected in November 2026 following the use of a Priority Review Voucher. A decision from the European Commission is expected in the second half of 2027.
Approval of additional drugs will broaden its diverse portfolio.
View the Zacks #1 Rank List
Image: Bigstock
Regeneron Secures FDA Approval for Rare Genetic Disorder Drug
Key Takeaways
Regeneron Pharmaceuticals, Inc. (REGN - Free Report) announced that the FDA has approved Pasatru (garetosmab-grts) to reduce the formation of new heterotopic ossification (HO) lesions and clinician-assessed flare-ups in adults with fibrodysplasia ossificans progressiva (FOP).
FOP is an ultra-rare genetic disorder characterized by progressive formation of abnormal bone within muscles, tendons, ligaments and other connective tissues, a process known as HO.
Pasatru is a fully human monoclonal antibody derived using Regeneron’s VelocImmune technology. It works by blocking Activin A, a protein discovered by Regeneron scientists to play a critical role in the development of HO lesions in people with FOP.
Regeneron’s shares have gained 8.9% so far this year compared with the industry’s 7% growth.
Image Source: Zacks Investment Research
More on REGN’s Pasatru Approval
The approval was based on results from the OPTIMA study demonstrating a 90% or greater reduction in new HO lesions at 56 weeks with a dramatic reduction in clinician-assessed flare-ups in adults with FOP.
HO affecting the jaw, spine, hips and rib cage can make essential activities such as speaking, eating, walking and breathing increasingly difficult, ultimately resulting in severe and progressive loss of mobility.
FOP affects approximately 900 people worldwide, with most patients becoming wheelchair-dependent by age 30 and having a median survival of around 56 years.
Given the severe mobility challenges associated with FOP, Pasatru may be administered in various care settings, including at-home infusion when appropriate. The recommended starting dose is 10 mg/kg, administered intravenously more than 60 minutes once every four weeks. For patients who do not tolerate the starting dose, treatment may be reduced to three mg/kg on the same monthly schedule.
Meanwhile, a phase III study of Pasatru in adolescents and children with FOP, OPTIMA 2, is planned to begin later this year.
In the European Union (EU), Pasatru is currently under regulatory review by the European Medicines Agency (EMA), with additional submissions planned in other markets, including Japan. The drug has previously received FDA Fast Track and Orphan Drug designations, along with Orphan Designation from the EMA and Japan’s Ministry of Health, Labour and Welfare.
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, while Bayer records the same outside the country.
Apart from Eylea, profits from the sales of asthma drug Dupixent are a primary growth driver for REGN.
Regeneron Pharmaceuticals, Inc. Price, Consensus and EPS Surprise
Regeneron Pharmaceuticals, Inc. price-consensus-eps-surprise-chart | Regeneron Pharmaceuticals, Inc. Quote
Regeneron has a collaboration agreement with Sanofi (SNY - Free Report) for drugs like Dupixent and Kevzara.
While SNY 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 gaining momentum, driven by Libtayo.
Meanwhile, the FDA earlier granted accelerated approval to Otarmeni (lunsotogene parvec, formerly DB-OTO), the first gene therapy cleared under the FDA Commissioner’s National Priority Voucher Program. The adeno-associated virus-based treatment is indicated for pediatric and adult patients with severe-to-profound hearing loss caused by OTOF gene variants. A regulatory filing for the therapy is currently under review in the EU for the treatment of biallelic OTOF variant-associated hearing loss.
In June 2026, the FDA and EMA accepted Regeneron’s regulatory applications for cemdisiran, a C5-targeting RNAi therapy, for the treatment of adults with generalized myasthenia gravis. The FDA granted priority review to the filing, with a decision expected in November 2026 following the use of a Priority Review Voucher. A decision from the European Commission is expected in the second half of 2027.
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.