Notizia - Recent Pharma, Healthcare and Biotech Happenings
Jun 17, 2025
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Celltrion, Inc. has announced that the FDA has approved an additional presentation of STEQEYMA (ustekinumab-stba), a biosimilar to STELARA, in a 45mg/0.5mL single-dose vial for subcutaneous injection. This new formulation is now indicated for pediatric patients aged 6 to 17 years weighing under 60kg with plaque psoriasis (PsO) or psoriatic arthritis (PsA). With this approval, STEQEYMA now mirrors all dosage forms and strengths of its reference product, STELARA, expanding treatment flexibility and continuity for younger patient populations.
“The new dosage form and strength of STEQEYMA allow us to better meet the specific needs of young patients, giving physicians a valuable treatment option with flexibility, supported by a well-established safety and efficacy profile,” said Hetal Patel, PharmD MBA, Vice President of Medical Affairs at Celltrion USA.
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Celltrion had previously received FDA approval in December 2024 for other formulations of STEQEYMA, including 45mg/0.5mL and 90mg/mL in single-dose prefilled syringes for subcutaneous injection, and 130mg/26mL in a single-dose vial for intravenous infusion. These were approved for both adult and pediatric patients aged 6 and above with PsO and PsA, and for adults with Crohn’s disease and ulcerative colitis.
“This approval reinforces our commitment to broadening access for all patient populations, including children aged 6 years and older living with chronic inflammatory conditions,” added Thomas Nusbickel, Chief Commercial Officer at Celltrion USA. “As a company with a strong legacy in immunology, we are dedicated to ensuring broader access and flexibility in care for patients of all ages.”
The approval was supported by robust Phase III clinical data, which confirmed biosimilarity between STEQEYMA and STELARA in terms of efficacy, safety, and immunogenicity. The FDA has also granted STEQEYMA full interchangeability with STELARA across all approved indications, following the expiration of exclusivity for the first interchangeable biosimilar in April 2025.
CSL has announced that the FDA has approved ANDEMBRY (garadacimab-gxii), making it the first and only prophylactic treatment for hereditary angioedema (HAE) that targets Factor XIIa. The once-monthly subcutaneous therapy is approved for use in both adult and pediatric patients aged 12 and older, offering rapid, self-administered protection from HAE attacks with a citrate-free formula delivered in 15 seconds or less via autoinjector.
“ANDEMBRY, the first monoclonal antibody discovered and developed entirely by CSL, offers people living with this life-threatening condition long-term control over their disease along with a convenient administration method,” said Dr. Bill Mezzanotte, Executive VP and Head of R&D at CSL. “This approval reflects CSL’s enduring commitment to addressing unmet needs in rare disease communities, including those living with HAE.”
The FDA’s approval was based on results from the pivotal Phase III VANGUARD trial published in The Lancet (April 2023). The study showed ANDEMBRY reduced HAE attacks by a median of over 99% and a least squares mean of 89.2% compared to placebo. Additionally, 62% of patients treated with ANDEMBRY remained attack-free during the treatment period. The therapy also significantly reduced moderate-to-severe attacks and the need for on-demand rescue medication. Safety data from the ongoing open-label extension study reaffirmed its favorable long-term safety profile, with injection-site reactions and mild adverse events such as nasopharyngitis being the most common.
“Many patients with HAE still face the burden of painful, life-threatening attacks and frequent injections,” said Dr. Tim Craig, Professor at Penn State University. “ANDEMBRY introduces a novel approach by inhibiting Factor XIIa, the top of the HAE cascade, which is a meaningful advance in how we manage this condition.”
Anthony J. Castaldo, CEO of the US HAE Association and HAE International, added, “A once-monthly subcutaneous therapy like ANDEMBRY provides our community with greater freedom and the opportunity to live life more fully.”
The FDA’s nod follows recent approvals of ANDEMBRY in Australia, the UK, EU, Japan, Switzerland, and the UAE, signaling a major global expansion of CSL’s HAE portfolio and the company’s continued leadership in rare immunological conditions.
Ocugen, Inc. has announced FDA clearance of its Investigational New Drug (IND) amendment to initiate a pivotal Phase II/III confirmatory trial of OCU410ST, a modifier gene therapy candidate developed for Stargardt disease and other ABCA4-associated retinopathies. The announcement follows the therapy’s Rare Pediatric Disease and Orphan Drug designations, reinforcing OCU410ST’s potential for treating rare, inherited retinal conditions for which no approved treatments currently exist.
“We’ve had a highly productive engagement with the FDA’s Center for Biologics Evaluation and Research,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “There is a visible sense of urgency from the agency in getting treatments to patients with no current options. With this clearance, we’re able to accelerate clinical development of OCU410ST by two to three years, bringing hope to those affected by this devastating condition.”
Positive data from Ocugen’s Phase I GARDian trial showed a favorable safety profile and promising efficacy signals, including a statistically significant two-line improvement in best corrected visual acuity (BCVA) and a 48% slower lesion growth at 12 months in treated eyes versus untreated controls. The new Phase II/III trial will enroll 51 patients with Stargardt disease, assigning 34 to receive a one-time subretinal injection of OCU410ST and 17 to a control group. The primary endpoint will focus on reduction in atrophic lesion size, while secondary endpoints include improvements in BCVA and low luminance visual acuity (LLVA).
“The launch of this pivotal trial marks a critical step in advancing our mission to deliver life-changing genetic therapies,” said Dr. Huma Qamar, Ocugen’s Chief Medical Officer. “OCU410ST, developed through our proprietary modifier gene therapy platform, aims to address the disease at its root, offering hope for lasting impact in pediatric and adult patients.”
With approximately 1 million people worldwide affected by Stargardt disease and ABCA4 retinopathies, the trial addresses a significant unmet need. “This study is scientifically robust and patient-centered,” said Dr. Lejla Vajzovic, Professor of Ophthalmology at Duke University. “We’re hopeful it will bring us closer to an effective therapy for children and young adults battling this progressive condition.”
Ocugen plans to submit a Biologics License Application (BLA) for OCU410ST in 2027, aligning with its roadmap to file three BLAs over the next three years.
Milestone Pharmaceuticals has submitted its formal response to the FDA addressing the Complete Response Letter (CRL) for its New Drug Application (NDA) for CARDAMYST (etripamil), a nasal spray intended for the acute treatment of paroxysmal supraventricular tachycardia (PSVT) in adults. The submission follows a productive Type A meeting with the FDA, during which the agency provided guidance enabling Milestone to promptly finalize and file its response.
“We appreciate the constructive dialogue with the FDA and the clarity provided during our recent Type A meeting,” said Joe Oliveto, President and CEO of Milestone Pharmaceuticals. “With this resubmission, we are optimistic about a potential PDUFA date later this year and the opportunity to deliver CARDAMYST to patients.”
If approved, CARDAMYST would become the first and only self-administered treatment for the rapid resolution of PSVT episodes—a breakthrough for patients who currently rely on emergency room visits or intravenous therapies. The company expects the FDA to determine the acceptance and classification of the response within 30 days, setting the stage for a review period of either two or six months.
AbbVie has announced that the FDA has approved a major label expansion for MAVYRET (glecaprevir/pibrentasvir), making it the first and only direct-acting antiviral (DAA) therapy approved for treating acute hepatitis C virus (HCV) in both adults and pediatric patients aged three years and older. The once-daily oral treatment, previously indicated for chronic HCV, can now be prescribed for acute cases, offering an 8-week regimen with a 96% cure rate.
“MAVYRET has treated more than one million patients with HCV, but we recognize that a significant need remains for patients with acute infection,” said Roopal Thakkar, M.D., Executive Vice President, R&D and Chief Scientific Officer at AbbVie. “This label expansion, combined with test-and-treat models of care, helps support broader access to treatment and brings us closer to achieving the global hepatitis C elimination goals.”
Acute HCV often presents without symptoms and, if untreated, may lead to serious complications such as cirrhosis and liver cancer. The U.S. alone is projected to face over $120 billion in medical costs by 2035 due to untreated chronic liver conditions. By enabling earlier intervention, MAVYRET could significantly reduce this burden.
“The physical, emotional, and economic burden of a curable condition like hepatitis C is far too great,” added Dr. John Ward, Director of the Coalition for Global Hepatitis Elimination. “With safe, effective therapies like MAVYRET now available for early-stage treatment, we can stop hepatitis C before it becomes a deadly, chronic disease. No one should die from hepatitis C.”
The FDA’s decision was supported by results from a Phase III, multicenter study evaluating MAVYRET in acute HCV patients, where the majority of adverse events were mild to moderate. The approval follows Breakthrough Therapy Designation granted to MAVYRET, recognizing its potential to significantly improve care for patients with serious viral infections.
Article in PDF
Jun 17, 2025
Table of Contents
Celltrion, Inc. has announced that the FDA has approved an additional presentation of STEQEYMA (ustekinumab-stba), a biosimilar to STELARA, in a 45mg/0.5mL single-dose vial for subcutaneous injection. This new formulation is now indicated for pediatric patients aged 6 to 17 years weighing under 60kg with plaque psoriasis (PsO) or psoriatic arthritis (PsA). With this approval, STEQEYMA now mirrors all dosage forms and strengths of its reference product, STELARA, expanding treatment flexibility and continuity for younger patient populations.
“The new dosage form and strength of STEQEYMA allow us to better meet the specific needs of young patients, giving physicians a valuable treatment option with flexibility, supported by a well-established safety and efficacy profile,” said Hetal Patel, PharmD MBA, Vice President of Medical Affairs at Celltrion USA.
Celltrion had previously received FDA approval in December 2024 for other formulations of STEQEYMA, including 45mg/0.5mL and 90mg/mL in single-dose prefilled syringes for subcutaneous injection, and 130mg/26mL in a single-dose vial for intravenous infusion. These were approved for both adult and pediatric patients aged 6 and above with PsO and PsA, and for adults with Crohn’s disease and ulcerative colitis.
“This approval reinforces our commitment to broadening access for all patient populations, including children aged 6 years and older living with chronic inflammatory conditions,” added Thomas Nusbickel, Chief Commercial Officer at Celltrion USA. “As a company with a strong legacy in immunology, we are dedicated to ensuring broader access and flexibility in care for patients of all ages.”
The approval was supported by robust Phase III clinical data, which confirmed biosimilarity between STEQEYMA and STELARA in terms of efficacy, safety, and immunogenicity. The FDA has also granted STEQEYMA full interchangeability with STELARA across all approved indications, following the expiration of exclusivity for the first interchangeable biosimilar in April 2025.
CSL has announced that the FDA has approved ANDEMBRY (garadacimab-gxii), making it the first and only prophylactic treatment for hereditary angioedema (HAE) that targets Factor XIIa. The once-monthly subcutaneous therapy is approved for use in both adult and pediatric patients aged 12 and older, offering rapid, self-administered protection from HAE attacks with a citrate-free formula delivered in 15 seconds or less via autoinjector.
“ANDEMBRY, the first monoclonal antibody discovered and developed entirely by CSL, offers people living with this life-threatening condition long-term control over their disease along with a convenient administration method,” said Dr. Bill Mezzanotte, Executive VP and Head of R&D at CSL. “This approval reflects CSL’s enduring commitment to addressing unmet needs in rare disease communities, including those living with HAE.”
The FDA’s approval was based on results from the pivotal Phase III VANGUARD trial published in The Lancet (April 2023). The study showed ANDEMBRY reduced HAE attacks by a median of over 99% and a least squares mean of 89.2% compared to placebo. Additionally, 62% of patients treated with ANDEMBRY remained attack-free during the treatment period. The therapy also significantly reduced moderate-to-severe attacks and the need for on-demand rescue medication. Safety data from the ongoing open-label extension study reaffirmed its favorable long-term safety profile, with injection-site reactions and mild adverse events such as nasopharyngitis being the most common.
“Many patients with HAE still face the burden of painful, life-threatening attacks and frequent injections,” said Dr. Tim Craig, Professor at Penn State University. “ANDEMBRY introduces a novel approach by inhibiting Factor XIIa, the top of the HAE cascade, which is a meaningful advance in how we manage this condition.”
Anthony J. Castaldo, CEO of the US HAE Association and HAE International, added, “A once-monthly subcutaneous therapy like ANDEMBRY provides our community with greater freedom and the opportunity to live life more fully.”
The FDA’s nod follows recent approvals of ANDEMBRY in Australia, the UK, EU, Japan, Switzerland, and the UAE, signaling a major global expansion of CSL’s HAE portfolio and the company’s continued leadership in rare immunological conditions.
Ocugen, Inc. has announced FDA clearance of its Investigational New Drug (IND) amendment to initiate a pivotal Phase II/III confirmatory trial of OCU410ST, a modifier gene therapy candidate developed for Stargardt disease and other ABCA4-associated retinopathies. The announcement follows the therapy’s Rare Pediatric Disease and Orphan Drug designations, reinforcing OCU410ST’s potential for treating rare, inherited retinal conditions for which no approved treatments currently exist.
“We’ve had a highly productive engagement with the FDA’s Center for Biologics Evaluation and Research,” said Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen. “There is a visible sense of urgency from the agency in getting treatments to patients with no current options. With this clearance, we’re able to accelerate clinical development of OCU410ST by two to three years, bringing hope to those affected by this devastating condition.”
Positive data from Ocugen’s Phase I GARDian trial showed a favorable safety profile and promising efficacy signals, including a statistically significant two-line improvement in best corrected visual acuity (BCVA) and a 48% slower lesion growth at 12 months in treated eyes versus untreated controls. The new Phase II/III trial will enroll 51 patients with Stargardt disease, assigning 34 to receive a one-time subretinal injection of OCU410ST and 17 to a control group. The primary endpoint will focus on reduction in atrophic lesion size, while secondary endpoints include improvements in BCVA and low luminance visual acuity (LLVA).
“The launch of this pivotal trial marks a critical step in advancing our mission to deliver life-changing genetic therapies,” said Dr. Huma Qamar, Ocugen’s Chief Medical Officer. “OCU410ST, developed through our proprietary modifier gene therapy platform, aims to address the disease at its root, offering hope for lasting impact in pediatric and adult patients.”
With approximately 1 million people worldwide affected by Stargardt disease and ABCA4 retinopathies, the trial addresses a significant unmet need. “This study is scientifically robust and patient-centered,” said Dr. Lejla Vajzovic, Professor of Ophthalmology at Duke University. “We’re hopeful it will bring us closer to an effective therapy for children and young adults battling this progressive condition.”
Ocugen plans to submit a Biologics License Application (BLA) for OCU410ST in 2027, aligning with its roadmap to file three BLAs over the next three years.
Milestone Pharmaceuticals has submitted its formal response to the FDA addressing the Complete Response Letter (CRL) for its New Drug Application (NDA) for CARDAMYST (etripamil), a nasal spray intended for the acute treatment of paroxysmal supraventricular tachycardia (PSVT) in adults. The submission follows a productive Type A meeting with the FDA, during which the agency provided guidance enabling Milestone to promptly finalize and file its response.
“We appreciate the constructive dialogue with the FDA and the clarity provided during our recent Type A meeting,” said Joe Oliveto, President and CEO of Milestone Pharmaceuticals. “With this resubmission, we are optimistic about a potential PDUFA date later this year and the opportunity to deliver CARDAMYST to patients.”
If approved, CARDAMYST would become the first and only self-administered treatment for the rapid resolution of PSVT episodes—a breakthrough for patients who currently rely on emergency room visits or intravenous therapies. The company expects the FDA to determine the acceptance and classification of the response within 30 days, setting the stage for a review period of either two or six months.
AbbVie has announced that the FDA has approved a major label expansion for MAVYRET (glecaprevir/pibrentasvir), making it the first and only direct-acting antiviral (DAA) therapy approved for treating acute hepatitis C virus (HCV) in both adults and pediatric patients aged three years and older. The once-daily oral treatment, previously indicated for chronic HCV, can now be prescribed for acute cases, offering an 8-week regimen with a 96% cure rate.
“MAVYRET has treated more than one million patients with HCV, but we recognize that a significant need remains for patients with acute infection,” said Roopal Thakkar, M.D., Executive Vice President, R&D and Chief Scientific Officer at AbbVie. “This label expansion, combined with test-and-treat models of care, helps support broader access to treatment and brings us closer to achieving the global hepatitis C elimination goals.”
Acute HCV often presents without symptoms and, if untreated, may lead to serious complications such as cirrhosis and liver cancer. The U.S. alone is projected to face over $120 billion in medical costs by 2035 due to untreated chronic liver conditions. By enabling earlier intervention, MAVYRET could significantly reduce this burden.
“The physical, emotional, and economic burden of a curable condition like hepatitis C is far too great,” added Dr. John Ward, Director of the Coalition for Global Hepatitis Elimination. “With safe, effective therapies like MAVYRET now available for early-stage treatment, we can stop hepatitis C before it becomes a deadly, chronic disease. No one should die from hepatitis C.”
The FDA’s decision was supported by results from a Phase III, multicenter study evaluating MAVYRET in acute HCV patients, where the majority of adverse events were mild to moderate. The approval follows Breakthrough Therapy Designation granted to MAVYRET, recognizing its potential to significantly improve care for patients with serious viral infections.
