Notizia - Recent Pharma, Healthcare and Biotech Happenings
Aug 12, 2025
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Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd., announced that the FDA has approved AJOVY (fremanezumab-vfrm) for the preventive treatment of episodic migraine in children and adolescents aged 6–17 years who weigh at least 45 kg (99 lbs). This approval makes AJOVY the first and only calcitonin gene-related peptide (CGRP) antagonist indicated for both pediatric episodic migraine prevention and migraine prevention in adults, expanding access across age groups.
“Migraines are a common yet invisible condition that can severely disrupt daily life for children and adolescents,” said Chris Fox, Executive Vice President, U.S. Commercial and Innovative Franchise Lead and Head of Global Marketing Business at Teva. “With this FDA approval, AJOVY now offers younger patients a new treatment option, addressing a long-standing gap in care and offering families added support as they navigate the challenges of this condition.”
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AJOVY, already approved in the U.S. since 2018 for adults, is administered once monthly and can be given in-office or at home. The single-dose injection offers a practical option intended to improve adherence and reduce treatment burden for families managing pediatric migraine—a condition affecting an estimated 1 in 10 children and adolescents in the U.S., often resulting in missed school days and social disruption.
“Pediatric migraine is a complex condition that can significantly impact a child’s daily life, from school performance to emotional well-being,” said Dr. Jennifer McVige, Pediatric Neurologist at the DENT Neurologic Institute. “Having an FDA-approved treatment like AJOVY provides a targeted preventive approach that can help reduce the frequency of attacks in younger patients and help clinicians better manage this often-overlooked condition.”
NRx Pharmaceuticals, Inc. announced that the FDA has granted Fast Track designation to NRX-100 for the treatment of suicidal ideation in patients with depression, including bipolar depression. This decision expands the drug’s addressable population tenfold compared to its 2017 designation, which covered only suicidal bipolar depression when used in combination with NRX-101. The designation also aligns NRX-100 with eligibility for the FDA’s Accelerated Approval and the Commissioner’s National Priority Voucher (CNPV) programs.
“We thank FDA for its thoughtful review of our Fast Track designation request, and believe this regulatory determination is a significant step forward in our goal to address the national crisis of suicide among soldiers, first responders, veterans, and civilians alike,” said Dr. Jonathan Javitt, Chairman and CEO of NRx Pharmaceuticals. “Large-scale government-supported trials have demonstrated a robust and statistically significant reduction in suicidal ideation and depression with administration of ketamine.”
The FDA’s decision was supported by multiple controlled trials showing NRX-100’s significant impact on reducing suicidal ideation. In a Columbia University study, intravenous ketamine achieved a 55% response rate compared to 30% for the active comparator, while a French government-sponsored trial showed a 63% full remission rate within three days versus 31% with placebo. Unlike intranasal forms, these effects were achieved with intravenous administration.
Under the Fast Track program, NRx plans to post an expanded access policy for NRX-100 within two weeks and request a meeting with FDA leadership to finalize its Accelerated Approval/CNPV submission. The designation grants the company benefits such as enhanced FDA communication, rolling review, and potential priority review, potentially accelerating access for the estimated 13 million Americans who consider suicide each year.
Boehringer Ingelheim announced that the FDA has granted accelerated approval to HERNEXEOS (zongertinib tablets) for the treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) harboring HER2 (ERBB2) tyrosine kinase domain activating mutations, as confirmed by an FDA-approved test, and who have received prior systemic therapy. The approval is based on objective response rate (ORR) and duration of response (DoR) data, with continued approval contingent upon confirmatory trial results.
“With the approval of zongertinib, we have an effective, targeted, orally administered treatment option for patients with HER2-mutant non-small cell lung cancer in the U.S. that not only elicits a durable response but, importantly, has a manageable safety profile,” said Dr. John Heymach, MD, PhD, chair of Thoracic/Head and Neck Medical Oncology at The University of Texas MD Anderson Cancer Center and coordinating investigator for the Beamion-LUNG 1 trial.
The FDA’s decision follows results from the Phase Ib Beamion-LUNG 1 trial, where HERNEXEOS achieved a 75% ORR (N=71), including 6% complete responses and 69% partial responses, with a DoR of ≥6 months in 58% of patients. The therapy showed a manageable safety profile with a 2.9% discontinuation rate; the most common adverse reactions included diarrhea, hepatotoxicity, rash, fatigue, and nausea.
“We are grateful to bring forward HERNEXEOS, which has the potential to reset the benchmark for those living with HER2-mutant advanced NSCLC, a condition associated with a particularly poor prognosis,” said Shashank Deshpande, Chairman of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “Recognizing its potential, we accelerated development to deliver this treatment to patients within four years of starting the first clinical trial.”
Genmab A/S announced that the Phase III EPCORE® FL-1 trial of subcutaneous epcoritamab in combination with rituximab and lenalidomide (R2) met its dual primary endpoints in adult patients with relapsed or refractory (R/R) follicular lymphoma (FL). The regimen significantly improved overall response rate (ORR) and progression-free survival (PFS), reducing the risk of disease progression or death by 79% compared to R2 alone. Data from this pre-planned interim analysis will be presented at the American Society of Hematology (ASH) Annual Meeting and will form the basis for global regulatory submissions.
“While therapeutic options exist for patients with relapsed or refractory follicular lymphoma, response rates tend to decline and durability diminishes with each subsequent line of treatment,” said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. “The results from this trial, and the FDA’s acceptance of our sBLA for priority review, demonstrate the potential of this epcoritamab combination therapy to reshape the treatment landscape.”
The FDA has accepted for priority review a supplemental Biologics License Application (sBLA) for epcoritamab plus R2 following at least one prior systemic therapy, with a target action date of November 30, 2025. The application is supported by data showing an ORR of 95.7% and a hazard ratio for PFS of 0.21 in the intent-to-treat population. If approved, it would become the first bispecific antibody combination regimen available in the U.S. as a second-line option for R/R FL.
The safety profile of the combination was consistent with known profiles of epcoritamab and R2, with no new safety signals observed. Epcoritamab is already approved in the U.S. as a single agent for R/R FL after two or more prior systemic therapies and has received Breakthrough Therapy Designation in combination with R2 for this indication.
Tiziana Life Sciences, Ltd., announced that the FDA has approved its Investigational New Drug (IND) application for a phase IIa trial evaluating intranasal foralumab in patients with Multiple System Atrophy (MSA), a rare and rapidly progressive neurodegenerative disorder with no approved treatments. The six-month, open-label study (NCT06868628) will assess foralumab’s effects on microglial activation, clinical outcomes, and safety in MSA patients through eight 3-week dosing cycles.
“We are pleased that the FDA has approved the IND to treat MSA patients with nasal foralumab,” said Vikram Khurana, MD, PhD, Division Chief of Movement Disorders and Director of the MSA Center of Excellence at Brigham and Women’s Hospital. “Neuroinflammation plays a significant role in MSA’s rapid progression. Intranasal foralumab offers a novel therapeutic pathway by targeting T-cell mediated immune activation in the brain.”
Foralumab, a fully human anti-CD3 monoclonal antibody, is designed to induce regulatory T cells and modulate T-cell-driven inflammation, potentially slowing neuronal damage in neurodegenerative diseases. The therapy’s intranasal delivery aims to engage immune regulation while minimizing systemic immune suppression.
Ivor Elrifi, CEO of Tiziana Life Sciences, added, “Its ability to reduce microglial activation presents a promising therapeutic strategy for MSA as well as other neuroinflammatory diseases such as Multiple Sclerosis, Alzheimer’s Disease, and ALS. This approach could represent a meaningful step toward addressing significant unmet medical needs in these patient populations.”
Article in PDF
Aug 12, 2025
Table of Contents
Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd., announced that the FDA has approved AJOVY (fremanezumab-vfrm) for the preventive treatment of episodic migraine in children and adolescents aged 6–17 years who weigh at least 45 kg (99 lbs). This approval makes AJOVY the first and only calcitonin gene-related peptide (CGRP) antagonist indicated for both pediatric episodic migraine prevention and migraine prevention in adults, expanding access across age groups.
“Migraines are a common yet invisible condition that can severely disrupt daily life for children and adolescents,” said Chris Fox, Executive Vice President, U.S. Commercial and Innovative Franchise Lead and Head of Global Marketing Business at Teva. “With this FDA approval, AJOVY now offers younger patients a new treatment option, addressing a long-standing gap in care and offering families added support as they navigate the challenges of this condition.”
AJOVY, already approved in the U.S. since 2018 for adults, is administered once monthly and can be given in-office or at home. The single-dose injection offers a practical option intended to improve adherence and reduce treatment burden for families managing pediatric migraine—a condition affecting an estimated 1 in 10 children and adolescents in the U.S., often resulting in missed school days and social disruption.
“Pediatric migraine is a complex condition that can significantly impact a child’s daily life, from school performance to emotional well-being,” said Dr. Jennifer McVige, Pediatric Neurologist at the DENT Neurologic Institute. “Having an FDA-approved treatment like AJOVY provides a targeted preventive approach that can help reduce the frequency of attacks in younger patients and help clinicians better manage this often-overlooked condition.”
NRx Pharmaceuticals, Inc. announced that the FDA has granted Fast Track designation to NRX-100 for the treatment of suicidal ideation in patients with depression, including bipolar depression. This decision expands the drug’s addressable population tenfold compared to its 2017 designation, which covered only suicidal bipolar depression when used in combination with NRX-101. The designation also aligns NRX-100 with eligibility for the FDA’s Accelerated Approval and the Commissioner’s National Priority Voucher (CNPV) programs.
“We thank FDA for its thoughtful review of our Fast Track designation request, and believe this regulatory determination is a significant step forward in our goal to address the national crisis of suicide among soldiers, first responders, veterans, and civilians alike,” said Dr. Jonathan Javitt, Chairman and CEO of NRx Pharmaceuticals. “Large-scale government-supported trials have demonstrated a robust and statistically significant reduction in suicidal ideation and depression with administration of ketamine.”
The FDA’s decision was supported by multiple controlled trials showing NRX-100’s significant impact on reducing suicidal ideation. In a Columbia University study, intravenous ketamine achieved a 55% response rate compared to 30% for the active comparator, while a French government-sponsored trial showed a 63% full remission rate within three days versus 31% with placebo. Unlike intranasal forms, these effects were achieved with intravenous administration.
Under the Fast Track program, NRx plans to post an expanded access policy for NRX-100 within two weeks and request a meeting with FDA leadership to finalize its Accelerated Approval/CNPV submission. The designation grants the company benefits such as enhanced FDA communication, rolling review, and potential priority review, potentially accelerating access for the estimated 13 million Americans who consider suicide each year.
Boehringer Ingelheim announced that the FDA has granted accelerated approval to HERNEXEOS (zongertinib tablets) for the treatment of adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) harboring HER2 (ERBB2) tyrosine kinase domain activating mutations, as confirmed by an FDA-approved test, and who have received prior systemic therapy. The approval is based on objective response rate (ORR) and duration of response (DoR) data, with continued approval contingent upon confirmatory trial results.
“With the approval of zongertinib, we have an effective, targeted, orally administered treatment option for patients with HER2-mutant non-small cell lung cancer in the U.S. that not only elicits a durable response but, importantly, has a manageable safety profile,” said Dr. John Heymach, MD, PhD, chair of Thoracic/Head and Neck Medical Oncology at The University of Texas MD Anderson Cancer Center and coordinating investigator for the Beamion-LUNG 1 trial.
The FDA’s decision follows results from the Phase Ib Beamion-LUNG 1 trial, where HERNEXEOS achieved a 75% ORR (N=71), including 6% complete responses and 69% partial responses, with a DoR of ≥6 months in 58% of patients. The therapy showed a manageable safety profile with a 2.9% discontinuation rate; the most common adverse reactions included diarrhea, hepatotoxicity, rash, fatigue, and nausea.
“We are grateful to bring forward HERNEXEOS, which has the potential to reset the benchmark for those living with HER2-mutant advanced NSCLC, a condition associated with a particularly poor prognosis,” said Shashank Deshpande, Chairman of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “Recognizing its potential, we accelerated development to deliver this treatment to patients within four years of starting the first clinical trial.”
Genmab A/S announced that the Phase III EPCORE® FL-1 trial of subcutaneous epcoritamab in combination with rituximab and lenalidomide (R2) met its dual primary endpoints in adult patients with relapsed or refractory (R/R) follicular lymphoma (FL). The regimen significantly improved overall response rate (ORR) and progression-free survival (PFS), reducing the risk of disease progression or death by 79% compared to R2 alone. Data from this pre-planned interim analysis will be presented at the American Society of Hematology (ASH) Annual Meeting and will form the basis for global regulatory submissions.
“While therapeutic options exist for patients with relapsed or refractory follicular lymphoma, response rates tend to decline and durability diminishes with each subsequent line of treatment,” said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab. “The results from this trial, and the FDA’s acceptance of our sBLA for priority review, demonstrate the potential of this epcoritamab combination therapy to reshape the treatment landscape.”
The FDA has accepted for priority review a supplemental Biologics License Application (sBLA) for epcoritamab plus R2 following at least one prior systemic therapy, with a target action date of November 30, 2025. The application is supported by data showing an ORR of 95.7% and a hazard ratio for PFS of 0.21 in the intent-to-treat population. If approved, it would become the first bispecific antibody combination regimen available in the U.S. as a second-line option for R/R FL.
The safety profile of the combination was consistent with known profiles of epcoritamab and R2, with no new safety signals observed. Epcoritamab is already approved in the U.S. as a single agent for R/R FL after two or more prior systemic therapies and has received Breakthrough Therapy Designation in combination with R2 for this indication.
Tiziana Life Sciences, Ltd., announced that the FDA has approved its Investigational New Drug (IND) application for a phase IIa trial evaluating intranasal foralumab in patients with Multiple System Atrophy (MSA), a rare and rapidly progressive neurodegenerative disorder with no approved treatments. The six-month, open-label study (NCT06868628) will assess foralumab’s effects on microglial activation, clinical outcomes, and safety in MSA patients through eight 3-week dosing cycles.
“We are pleased that the FDA has approved the IND to treat MSA patients with nasal foralumab,” said Vikram Khurana, MD, PhD, Division Chief of Movement Disorders and Director of the MSA Center of Excellence at Brigham and Women’s Hospital. “Neuroinflammation plays a significant role in MSA’s rapid progression. Intranasal foralumab offers a novel therapeutic pathway by targeting T-cell mediated immune activation in the brain.”
Foralumab, a fully human anti-CD3 monoclonal antibody, is designed to induce regulatory T cells and modulate T-cell-driven inflammation, potentially slowing neuronal damage in neurodegenerative diseases. The therapy’s intranasal delivery aims to engage immune regulation while minimizing systemic immune suppression.
Ivor Elrifi, CEO of Tiziana Life Sciences, added, “Its ability to reduce microglial activation presents a promising therapeutic strategy for MSA as well as other neuroinflammatory diseases such as Multiple Sclerosis, Alzheimer’s Disease, and ALS. This approach could represent a meaningful step toward addressing significant unmet medical needs in these patient populations.”
