May 20, 2025
Table of Contents
The FDA approved ZYNYZ (retifanlimab-dlwr), a PD-1 inhibitor developed by Incyte, for the treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). The approval includes two indications, in combination with carboplatin and paclitaxel chemotherapy as a first-line treatment, and as monotherapy for patients whose disease progressed on or who are intolerant to platinum-based therapy. This milestone is based on data from the Phase III POD1UM-303/InterAACT2 and Phase II POD1UM-202 trials, which demonstrated significant improvements in progression-free survival (PFS) and overall survival (OS) with the combination therapy and meaningful responses with monotherapy.
According to POD1UM-303/InterAACT2 results, the ZYNYZ-chemotherapy group showed a 37% reduction in risk of progression or death (P=0.0006) and achieved a median PFS of 9.3 months compared to 7.4 months in the placebo group. The trial also showed a 6.2-month improvement in OS (P=0.0273) at interim analysis. Meanwhile, the monotherapy study POD1UM-202 demonstrated a 14% objective response rate (ORR) and a 49% disease control rate. Safety profiles for both regimens aligned with known risks associated with PD-1 inhibitors, with serious adverse reactions occurring in 47% of patients on combination therapy and 40% on monotherapy.
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Incyte CEO Hervé Hoppenot commented, “The FDA approval of ZYNYZ marks a pivotal moment, bringing effective combination and monotherapy treatment options to patients with advanced anal cancer after decades of limited innovation. At Incyte, we focus our efforts where we can make the biggest impact for patients. I am proud of our scientists and development teams for their perseverance in delivering the first approved PD-1 inhibitor to U.S. patients with SCAC.”
Dr. Marwan Fakih of City of Hope added, “Patients with inoperable locally recurrent or metastatic anal cancer have historically faced poor five-year survival rates and limited treatment options. The POD1UM data highlight the potential of ZYNYZ to be a meaningful new option, and notably demonstrate that the addition of ZYNYZ to platinum-based chemotherapy significantly improves progression-free survival.” David Winterflood, CEO of the Anal Cancer Foundation, also remarked on the significance of the approval: “The approval of ZYNYZ marks a step forward for advanced SCAC treatment, brings attention to a long-overlooked condition with limited treatment options and offers patients whose anal cancer has returned or spread an option to treat their disease.”
Incyte has also expanded its regulatory efforts globally, with submissions for Marketing Authorization in Europe and a J-NDA in Japan now under review.
Amneal Pharmaceuticals, Inc. has received FDA approval for BREKIYA (dihydroergotamine mesylate) injection, marking a major milestone in the treatment of migraine and cluster headaches. BREKIYA is the first and only DHE autoinjector designed for the acute treatment of migraine with or without aura and cluster headaches in adults. Unlike traditional DHE therapies that require administration in hospitals via intravenous routes, BREKIYA is a ready-to-use, subcutaneous autoinjector that allows patients to self-administer their medication at home, offering convenience, faster relief, and greater independence. The product is expected to be available in the second half of 2025 for appropriate patients.
The autoinjector provides sustained pain relief for those who experience inadequate response to oral therapies or deal with nausea, vomiting, or gastroparesis during attacks. Importantly, BREKIYA requires no refrigeration, assembly, or priming, and is administered via a single injection into the thigh. Given the limited treatment options for cluster headache, this new delivery format offers a promising solution for a widely underserved patient population.
Joe Renda, Senior Vice President and Chief Commercial Officer – Specialty at Amneal, emphasized the therapeutic innovation BREKIYA represents: “We are thrilled to offer the first and only ready-to-use autoinjector for patients suffering from acute migraine and cluster headaches. Physicians are familiar with DHE, which is an effective and well-established therapy that provides sustained relief for headaches. Our single-dose autoinjector represents an innovative therapeutic option for patients that allows for quick self-administration of the medication during these painful attacks without visiting the emergency room.”
With nearly 39 million Americans living with migraine and up to one million affected by cluster headaches, BREKIYA’s approval could significantly impact emergency room burden, where headache remains a top cause of visits. However, the use of BREKIYA comes with strict safety guidelines. Patients are advised not to take it alongside strong CYP3A4 inhibitors, and it is contraindicated in individuals with certain cardiovascular conditions, liver or kidney issues, and during pregnancy or breastfeeding. Proper medical guidance is essential before initiating treatment with BREKIYA.
Travere Therapeutics, Inc. announced that the FDA has accepted its supplemental New Drug Application (sNDA) for FILSPARI (sparsentan) for the treatment of focal segmental glomerulosclerosis (FSGS), a rare and serious kidney disorder. If approved, FILSPARI would become the first and only FDA-approved treatment for FSGS, which is one of the leading causes of kidney failure. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of January 13, 2026, and plans to convene an advisory committee to review the application.
“Today marks an important milestone in our mission to transform care for patients with rare kidney disease,” said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. “We are one step closer to potentially delivering the first approved treatment for people living with FSGS, a leading cause of kidney failure and devastating condition that urgently needs new treatment options.” He further added, “With this progress, we continue our commitment to the FSGS community who has been waiting so long for an effective medicine. We look forward to the upcoming review process.”
FILSPARI is a non-immunosuppressive, oral treatment designed to directly target podocyte injury by simultaneously blocking the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). It is currently approved for use in slowing kidney function decline in patients with IgA nephropathy. The application for FSGS is backed by strong clinical evidence from the Phase III DUPLEX Study and Phase II DUET Study, two of the most comprehensive interventional trials conducted in patients with FSGS. These studies showed FILSPARI’s superiority in reducing proteinuria compared to irbesartan, the current standard of care.
The clinical data not only confirmed the efficacy of FILSPARI in lowering protein levels in urine but also aligned with findings from the PARASOL workgroup, which demonstrated that long-term reduction in proteinuria significantly correlates with decreased risk of kidney failure. The drug was well-tolerated, and its safety profile was consistent with that of maximally dosed irbesartan, supporting its potential as a safe and effective new option for patients with FSGS.
AbbVie announced that the FDA has granted accelerated approval to EMRELIS (telisotuzumab vedotin-tllv) for the treatment of adult patients with locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) who have high c-Met protein overexpression and have received prior systemic therapy. EMRELIS is the first and only treatment approved for this specific patient population, which typically faces a poor prognosis and limited options.
High c-Met overexpression is defined by ≥ 50% of tumor cells showing strong (3+) staining, as determined by the FDA-approved Roche VENTANA® MET (SP44) RxDx Assay companion diagnostic. Approximately 25% of advanced EGFR wild-type, non-squamous NSCLC patients exhibit c-Met overexpression, with about half qualifying as high overexpressers.
EMRELIS is a c-Met-directed antibody-drug conjugate (ADC) designed to deliver a potent cytotoxic payload directly to c-Met-expressing tumor cells. The accelerated approval was based on data from the Phase II LUMINOSITY study, which demonstrated a 35% overall response rate (ORR) and a median duration of response (DOR) of 7.2 months in patients with high c-Met overexpression. The safety profile was manageable, with common adverse events including peripheral neuropathy, fatigue, decreased appetite, and peripheral edema.
“This approval represents a critical advancement in personalized lung cancer treatment for patients with high c-Met overexpressing NSCLC who have historically had limited options,” said Jonathan Goldman, MD, UCLA. AbbVie’s executive leadership highlighted EMRELIS as their first internally developed FDA-approved solid tumor therapy, emphasizing the company’s commitment to precision oncology.
Further confirmation of clinical benefit is being evaluated in the ongoing global Phase III TeliMET NSCLC-01 trial. The FDA also granted EMRELIS Breakthrough Therapy Designation in 2021 based on promising early data.
Lung cancer remains the leading cause of cancer-related mortality worldwide, underscoring the urgent need for new targeted therapies like EMRELIS to improve outcomes in difficult-to-treat populations.
Merck, known as MSD outside the U.S. and Canada, announced FDA approval of WELIREG (belzutifan), an oral hypoxia-inducible factor-2 alpha inhibitor, for the treatment of adult and pediatric patients aged 12 and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). PPGL are rare neuroendocrine tumors arising from similar tissues: pheochromocytomas originate in the adrenal gland, while paragangliomas develop outside the adrenal gland. These tumors may be driven by genetic syndromes or mutations, complicating diagnosis and management.
The approval is based on results from the single-arm Phase II LITESPARK-015 trial (NCT04924075), which enrolled 72 patients with measurable disease and controlled blood pressure. The primary endpoint was objective response rate (ORR) assessed by blinded independent central review (BICR) using RECIST v1.1 criteria. WELIREG was administered at 120 mg once daily until disease progression or unacceptable toxicity.
“This approval introduces the only approved non-surgical treatment option for patients with locally advanced or metastatic PPGL, addressing a critical unmet need in a rare and challenging disease area,” said Dr. Camilo Jimenez, MD Anderson Cancer Center.
Dr. Marjorie Green, Merck’s senior VP of oncology, highlighted that this represents the third FDA-approved indication for WELIREG, underscoring Merck’s commitment to innovative therapies for rare cancers. The WELIREG label includes a boxed warning for embryo-fetal toxicity; pregnancy must be ruled out before treatment, and effective non-hormonal contraception is required during therapy. WELIREG may cause severe anemia and hypoxia, necessitating careful monitoring and possible intervention.
Article in PDF
May 20, 2025
Table of Contents
The FDA approved ZYNYZ (retifanlimab-dlwr), a PD-1 inhibitor developed by Incyte, for the treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC). The approval includes two indications, in combination with carboplatin and paclitaxel chemotherapy as a first-line treatment, and as monotherapy for patients whose disease progressed on or who are intolerant to platinum-based therapy. This milestone is based on data from the Phase III POD1UM-303/InterAACT2 and Phase II POD1UM-202 trials, which demonstrated significant improvements in progression-free survival (PFS) and overall survival (OS) with the combination therapy and meaningful responses with monotherapy.
According to POD1UM-303/InterAACT2 results, the ZYNYZ-chemotherapy group showed a 37% reduction in risk of progression or death (P=0.0006) and achieved a median PFS of 9.3 months compared to 7.4 months in the placebo group. The trial also showed a 6.2-month improvement in OS (P=0.0273) at interim analysis. Meanwhile, the monotherapy study POD1UM-202 demonstrated a 14% objective response rate (ORR) and a 49% disease control rate. Safety profiles for both regimens aligned with known risks associated with PD-1 inhibitors, with serious adverse reactions occurring in 47% of patients on combination therapy and 40% on monotherapy.
Incyte CEO Hervé Hoppenot commented, “The FDA approval of ZYNYZ marks a pivotal moment, bringing effective combination and monotherapy treatment options to patients with advanced anal cancer after decades of limited innovation. At Incyte, we focus our efforts where we can make the biggest impact for patients. I am proud of our scientists and development teams for their perseverance in delivering the first approved PD-1 inhibitor to U.S. patients with SCAC.”
Dr. Marwan Fakih of City of Hope added, “Patients with inoperable locally recurrent or metastatic anal cancer have historically faced poor five-year survival rates and limited treatment options. The POD1UM data highlight the potential of ZYNYZ to be a meaningful new option, and notably demonstrate that the addition of ZYNYZ to platinum-based chemotherapy significantly improves progression-free survival.” David Winterflood, CEO of the Anal Cancer Foundation, also remarked on the significance of the approval: “The approval of ZYNYZ marks a step forward for advanced SCAC treatment, brings attention to a long-overlooked condition with limited treatment options and offers patients whose anal cancer has returned or spread an option to treat their disease.”
Incyte has also expanded its regulatory efforts globally, with submissions for Marketing Authorization in Europe and a J-NDA in Japan now under review.
Amneal Pharmaceuticals, Inc. has received FDA approval for BREKIYA (dihydroergotamine mesylate) injection, marking a major milestone in the treatment of migraine and cluster headaches. BREKIYA is the first and only DHE autoinjector designed for the acute treatment of migraine with or without aura and cluster headaches in adults. Unlike traditional DHE therapies that require administration in hospitals via intravenous routes, BREKIYA is a ready-to-use, subcutaneous autoinjector that allows patients to self-administer their medication at home, offering convenience, faster relief, and greater independence. The product is expected to be available in the second half of 2025 for appropriate patients.
The autoinjector provides sustained pain relief for those who experience inadequate response to oral therapies or deal with nausea, vomiting, or gastroparesis during attacks. Importantly, BREKIYA requires no refrigeration, assembly, or priming, and is administered via a single injection into the thigh. Given the limited treatment options for cluster headache, this new delivery format offers a promising solution for a widely underserved patient population.
Joe Renda, Senior Vice President and Chief Commercial Officer – Specialty at Amneal, emphasized the therapeutic innovation BREKIYA represents: “We are thrilled to offer the first and only ready-to-use autoinjector for patients suffering from acute migraine and cluster headaches. Physicians are familiar with DHE, which is an effective and well-established therapy that provides sustained relief for headaches. Our single-dose autoinjector represents an innovative therapeutic option for patients that allows for quick self-administration of the medication during these painful attacks without visiting the emergency room.”
With nearly 39 million Americans living with migraine and up to one million affected by cluster headaches, BREKIYA’s approval could significantly impact emergency room burden, where headache remains a top cause of visits. However, the use of BREKIYA comes with strict safety guidelines. Patients are advised not to take it alongside strong CYP3A4 inhibitors, and it is contraindicated in individuals with certain cardiovascular conditions, liver or kidney issues, and during pregnancy or breastfeeding. Proper medical guidance is essential before initiating treatment with BREKIYA.
Travere Therapeutics, Inc. announced that the FDA has accepted its supplemental New Drug Application (sNDA) for FILSPARI (sparsentan) for the treatment of focal segmental glomerulosclerosis (FSGS), a rare and serious kidney disorder. If approved, FILSPARI would become the first and only FDA-approved treatment for FSGS, which is one of the leading causes of kidney failure. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of January 13, 2026, and plans to convene an advisory committee to review the application.
“Today marks an important milestone in our mission to transform care for patients with rare kidney disease,” said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. “We are one step closer to potentially delivering the first approved treatment for people living with FSGS, a leading cause of kidney failure and devastating condition that urgently needs new treatment options.” He further added, “With this progress, we continue our commitment to the FSGS community who has been waiting so long for an effective medicine. We look forward to the upcoming review process.”
FILSPARI is a non-immunosuppressive, oral treatment designed to directly target podocyte injury by simultaneously blocking the endothelin A receptor (ETAR) and the angiotensin II subtype 1 receptor (AT1R). It is currently approved for use in slowing kidney function decline in patients with IgA nephropathy. The application for FSGS is backed by strong clinical evidence from the Phase III DUPLEX Study and Phase II DUET Study, two of the most comprehensive interventional trials conducted in patients with FSGS. These studies showed FILSPARI’s superiority in reducing proteinuria compared to irbesartan, the current standard of care.
The clinical data not only confirmed the efficacy of FILSPARI in lowering protein levels in urine but also aligned with findings from the PARASOL workgroup, which demonstrated that long-term reduction in proteinuria significantly correlates with decreased risk of kidney failure. The drug was well-tolerated, and its safety profile was consistent with that of maximally dosed irbesartan, supporting its potential as a safe and effective new option for patients with FSGS.
AbbVie announced that the FDA has granted accelerated approval to EMRELIS (telisotuzumab vedotin-tllv) for the treatment of adult patients with locally advanced or metastatic, non-squamous non-small cell lung cancer (NSCLC) who have high c-Met protein overexpression and have received prior systemic therapy. EMRELIS is the first and only treatment approved for this specific patient population, which typically faces a poor prognosis and limited options.
High c-Met overexpression is defined by ≥ 50% of tumor cells showing strong (3+) staining, as determined by the FDA-approved Roche VENTANA® MET (SP44) RxDx Assay companion diagnostic. Approximately 25% of advanced EGFR wild-type, non-squamous NSCLC patients exhibit c-Met overexpression, with about half qualifying as high overexpressers.
EMRELIS is a c-Met-directed antibody-drug conjugate (ADC) designed to deliver a potent cytotoxic payload directly to c-Met-expressing tumor cells. The accelerated approval was based on data from the Phase II LUMINOSITY study, which demonstrated a 35% overall response rate (ORR) and a median duration of response (DOR) of 7.2 months in patients with high c-Met overexpression. The safety profile was manageable, with common adverse events including peripheral neuropathy, fatigue, decreased appetite, and peripheral edema.
“This approval represents a critical advancement in personalized lung cancer treatment for patients with high c-Met overexpressing NSCLC who have historically had limited options,” said Jonathan Goldman, MD, UCLA. AbbVie’s executive leadership highlighted EMRELIS as their first internally developed FDA-approved solid tumor therapy, emphasizing the company’s commitment to precision oncology.
Further confirmation of clinical benefit is being evaluated in the ongoing global Phase III TeliMET NSCLC-01 trial. The FDA also granted EMRELIS Breakthrough Therapy Designation in 2021 based on promising early data.
Lung cancer remains the leading cause of cancer-related mortality worldwide, underscoring the urgent need for new targeted therapies like EMRELIS to improve outcomes in difficult-to-treat populations.
Merck, known as MSD outside the U.S. and Canada, announced FDA approval of WELIREG (belzutifan), an oral hypoxia-inducible factor-2 alpha inhibitor, for the treatment of adult and pediatric patients aged 12 and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). PPGL are rare neuroendocrine tumors arising from similar tissues: pheochromocytomas originate in the adrenal gland, while paragangliomas develop outside the adrenal gland. These tumors may be driven by genetic syndromes or mutations, complicating diagnosis and management.
The approval is based on results from the single-arm Phase II LITESPARK-015 trial (NCT04924075), which enrolled 72 patients with measurable disease and controlled blood pressure. The primary endpoint was objective response rate (ORR) assessed by blinded independent central review (BICR) using RECIST v1.1 criteria. WELIREG was administered at 120 mg once daily until disease progression or unacceptable toxicity.
“This approval introduces the only approved non-surgical treatment option for patients with locally advanced or metastatic PPGL, addressing a critical unmet need in a rare and challenging disease area,” said Dr. Camilo Jimenez, MD Anderson Cancer Center.
Dr. Marjorie Green, Merck’s senior VP of oncology, highlighted that this represents the third FDA-approved indication for WELIREG, underscoring Merck’s commitment to innovative therapies for rare cancers. The WELIREG label includes a boxed warning for embryo-fetal toxicity; pregnancy must be ruled out before treatment, and effective non-hormonal contraception is required during therapy. WELIREG may cause severe anemia and hypoxia, necessitating careful monitoring and possible intervention.
