Jul 29, 2025
Table of Contents
Ascendis Pharma A/S announced that the FDA has approved SKYTROFA (lonapegsomatropin-tcgd) for the treatment of adult growth hormone deficiency (GHD). Already approved for pediatric GHD since 2021, SKYTROFA is a once-weekly prodrug of somatropin designed to provide sustained release of unmodified human growth hormone, offering a less burdensome alternative to daily injections.
“This important milestone is the first of many planned label expansions supporting our goal to become the leading endocrinology rare disease company,” said Jan Mikkelsen, President and CEO of Ascendis Pharma. “We are pleased to expand SKYTROFA’s availability for adults initiating therapy or switching from existing treatment.”
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The FDA approval was based on positive data from the foresiGHt Phase III trial, a double-blind, placebo- and active-controlled study comparing weekly TransCon hGH with placebo and daily somatropin. Results demonstrated efficacy and safety benefits in adults with GHD.
“Adherence to daily injections remains a major issue in adult GHD,” noted Dr. Kevin Yuen, endocrinologist at Barrow Neurological Institute. “A once-weekly option like SKYTROFA may significantly improve both real-world adherence and patient outcomes, especially given the serious complications associated with this condition.”
SKYTROFA’s approval highlights a major advancement in the treatment landscape for adult GHD, addressing a key unmet need in long-term endocrine care.
Apellis Pharmaceuticals announced that the FDA has approved EMPAVELI (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 years and older to reduce proteinuria. These rare kidney diseases impact roughly 5,000 individuals in the U.S., often progressing to kidney failure without effective treatment.
“This approval is a pivotal moment,” said Dr. Carla Nester, lead investigator for the phase III VALIANT study and professor at the University of Iowa. “Patients, especially children, now have access to a highly effective therapy that significantly reduces proteinuria and helps preserve kidney function.”
The FDA’s decision is supported by positive data from the VALIANT study, where EMPAVELI demonstrated a 68% reduction in proteinuria, stabilization of kidney function, and clearance of C3 deposits. These benefits were observed across adolescent and adult populations, including those with post-transplant recurrence.
“EMPAVELI’s approval marks our third in four years and validates the potential of C3 inhibition to alter the course of rare diseases,” said Dr. Cedric Francois, CEO of Apellis. “We’re proud to bring this transformational therapy to patients and plan to advance its use in other renal indications.”
Josh Tarnoff, CEO of NephCure, added, “This is a historic milestone for the C3G and IC-MPGN communities. Apellis’ commitment to these underserved patients is commendable.”
PTC Therapeutics has received FDA approval for SEPHIENCE (sepiapterin) for the treatment of hyperphenylalaninemia (HPA) in both children and adults with sepiapterin-responsive phenylketonuria (PKU). The approval grants broad labeling for patients aged 1 month and older, marking a significant advancement in the treatment landscape for PKU.
“The broad labeling reflects the potential of SEPHIENCE to meet the significant unmet need of PKU patients,” said Dr. Matthew B. Klein, CEO of PTC Therapeutics. “Our experienced customer-facing teams are ready to bring this therapy to children and adults in the U.S. as quickly as possible.”
The FDA’s decision was supported by robust data from the phase III APHENITY trial and its long-term extension study, both of which demonstrated durable efficacy and safety. The approval also follows marketing authorization in the European Union, and regulatory reviews are underway in other major markets, including Japan and Brazil.
“This approval marks an exciting milestone for the PKU community,” said Catherine Warren, Executive Director of the National PKU Alliance. “We are eager to see the positive impact this new treatment option will have on advancing care and improving quality of life for patients of all ages and PKU subtypes.”
MAIA Biotechnology has received Fast Track designation from the FDA for its lead candidate, ateganosine (also known as THIO or 6-thio-dG), for the treatment of non-small cell lung cancer (NSCLC). The investigational first-in-class small molecule is currently being evaluated in the pivotal phase II THIO-101 trial, where it is administered prior to a checkpoint inhibitor.
“This is an important milestone for MAIA’s clinical development program,” said K. Robinson Lewis, Vice President and Head of Regulatory and Quality at MAIA. “Ateganosine has shown strong preclinical efficacy and encouraging survival data compared to current FDA-approved therapies for NSCLC.”
Designed to disrupt telomere structure and function in cancer cells, ateganosine works through telomerase-mediated activity to overcome resistance to immune checkpoint inhibitors. As of May 15, 2025, the median overall survival in the ongoing THIO-101 study was reported at 17.8 months, with the longest surviving patient reaching 24.3 months, far surpassing the historical benchmark of 5–6 months in similar NSCLC populations.
“If we are successful in this Fast Track pathway, ateganosine could receive accelerated approval and first-to-market status in a $34 billion NSCLC market,” noted Dr. Vlad Vitoc, Chairman and CEO of MAIA. “It has the potential to reshape how we approach NSCLC treatment and bring real hope to patients facing poor prognoses.”
Entera Bio Ltd. has secured alignment with the FDA for the design of its registrational phase III trial of EB613, marking a significant regulatory milestone for the company and the osteoporosis treatment landscape. In a written response to a Type A meeting request, the FDA agreed that a single, multinational, randomized, double-blind, placebo-controlled phase III study evaluating total hip bone mineral density (BMD) as the primary endpoint and vertebral fracture incidence as the key secondary endpoint would be sufficient to support a future New Drug Application (NDA).
“This regulatory update is a major milestone for Entera and the entire osteoporosis community,” stated Miranda Toledano, CEO of Entera Bio. “Our alignment with the FDA reflects the strength of our data and collaborative discussions. Importantly, it allows us to advance without waiting for the FDA’s qualification of SABRE, which is still expected later this year.”
This agreement departs from previous regulatory precedent, where phase III osteoporosis trials typically required fracture incidence as the primary endpoint, often extending trial timelines and raising cost and ethical hurdles. By accepting BMD as the primary endpoint, the FDA opens the door for a more streamlined path to innovation in osteoporosis drug development.
“Osteoporosis afflicts more women than heart attack, stroke, and breast cancer combined,” added Toledano. “With over 200 million women affected globally and few treatment innovations in recent years, EB613—our first-in-class oral, once-daily anabolic tablet—has the potential to address a significant unmet need by increasing skeletal mass, improving bone microarchitecture, and reducing fracture risk.”
Article in PDF
Jul 29, 2025
Table of Contents
Ascendis Pharma A/S announced that the FDA has approved SKYTROFA (lonapegsomatropin-tcgd) for the treatment of adult growth hormone deficiency (GHD). Already approved for pediatric GHD since 2021, SKYTROFA is a once-weekly prodrug of somatropin designed to provide sustained release of unmodified human growth hormone, offering a less burdensome alternative to daily injections.
“This important milestone is the first of many planned label expansions supporting our goal to become the leading endocrinology rare disease company,” said Jan Mikkelsen, President and CEO of Ascendis Pharma. “We are pleased to expand SKYTROFA’s availability for adults initiating therapy or switching from existing treatment.”
The FDA approval was based on positive data from the foresiGHt Phase III trial, a double-blind, placebo- and active-controlled study comparing weekly TransCon hGH with placebo and daily somatropin. Results demonstrated efficacy and safety benefits in adults with GHD.
“Adherence to daily injections remains a major issue in adult GHD,” noted Dr. Kevin Yuen, endocrinologist at Barrow Neurological Institute. “A once-weekly option like SKYTROFA may significantly improve both real-world adherence and patient outcomes, especially given the serious complications associated with this condition.”
SKYTROFA’s approval highlights a major advancement in the treatment landscape for adult GHD, addressing a key unmet need in long-term endocrine care.
Apellis Pharmaceuticals announced that the FDA has approved EMPAVELI (pegcetacoplan) for the treatment of C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) in patients aged 12 years and older to reduce proteinuria. These rare kidney diseases impact roughly 5,000 individuals in the U.S., often progressing to kidney failure without effective treatment.
“This approval is a pivotal moment,” said Dr. Carla Nester, lead investigator for the phase III VALIANT study and professor at the University of Iowa. “Patients, especially children, now have access to a highly effective therapy that significantly reduces proteinuria and helps preserve kidney function.”
The FDA’s decision is supported by positive data from the VALIANT study, where EMPAVELI demonstrated a 68% reduction in proteinuria, stabilization of kidney function, and clearance of C3 deposits. These benefits were observed across adolescent and adult populations, including those with post-transplant recurrence.
“EMPAVELI’s approval marks our third in four years and validates the potential of C3 inhibition to alter the course of rare diseases,” said Dr. Cedric Francois, CEO of Apellis. “We’re proud to bring this transformational therapy to patients and plan to advance its use in other renal indications.”
Josh Tarnoff, CEO of NephCure, added, “This is a historic milestone for the C3G and IC-MPGN communities. Apellis’ commitment to these underserved patients is commendable.”
PTC Therapeutics has received FDA approval for SEPHIENCE (sepiapterin) for the treatment of hyperphenylalaninemia (HPA) in both children and adults with sepiapterin-responsive phenylketonuria (PKU). The approval grants broad labeling for patients aged 1 month and older, marking a significant advancement in the treatment landscape for PKU.
“The broad labeling reflects the potential of SEPHIENCE to meet the significant unmet need of PKU patients,” said Dr. Matthew B. Klein, CEO of PTC Therapeutics. “Our experienced customer-facing teams are ready to bring this therapy to children and adults in the U.S. as quickly as possible.”
The FDA’s decision was supported by robust data from the phase III APHENITY trial and its long-term extension study, both of which demonstrated durable efficacy and safety. The approval also follows marketing authorization in the European Union, and regulatory reviews are underway in other major markets, including Japan and Brazil.
“This approval marks an exciting milestone for the PKU community,” said Catherine Warren, Executive Director of the National PKU Alliance. “We are eager to see the positive impact this new treatment option will have on advancing care and improving quality of life for patients of all ages and PKU subtypes.”
MAIA Biotechnology has received Fast Track designation from the FDA for its lead candidate, ateganosine (also known as THIO or 6-thio-dG), for the treatment of non-small cell lung cancer (NSCLC). The investigational first-in-class small molecule is currently being evaluated in the pivotal phase II THIO-101 trial, where it is administered prior to a checkpoint inhibitor.
“This is an important milestone for MAIA’s clinical development program,” said K. Robinson Lewis, Vice President and Head of Regulatory and Quality at MAIA. “Ateganosine has shown strong preclinical efficacy and encouraging survival data compared to current FDA-approved therapies for NSCLC.”
Designed to disrupt telomere structure and function in cancer cells, ateganosine works through telomerase-mediated activity to overcome resistance to immune checkpoint inhibitors. As of May 15, 2025, the median overall survival in the ongoing THIO-101 study was reported at 17.8 months, with the longest surviving patient reaching 24.3 months, far surpassing the historical benchmark of 5–6 months in similar NSCLC populations.
“If we are successful in this Fast Track pathway, ateganosine could receive accelerated approval and first-to-market status in a $34 billion NSCLC market,” noted Dr. Vlad Vitoc, Chairman and CEO of MAIA. “It has the potential to reshape how we approach NSCLC treatment and bring real hope to patients facing poor prognoses.”
Entera Bio Ltd. has secured alignment with the FDA for the design of its registrational phase III trial of EB613, marking a significant regulatory milestone for the company and the osteoporosis treatment landscape. In a written response to a Type A meeting request, the FDA agreed that a single, multinational, randomized, double-blind, placebo-controlled phase III study evaluating total hip bone mineral density (BMD) as the primary endpoint and vertebral fracture incidence as the key secondary endpoint would be sufficient to support a future New Drug Application (NDA).
“This regulatory update is a major milestone for Entera and the entire osteoporosis community,” stated Miranda Toledano, CEO of Entera Bio. “Our alignment with the FDA reflects the strength of our data and collaborative discussions. Importantly, it allows us to advance without waiting for the FDA’s qualification of SABRE, which is still expected later this year.”
This agreement departs from previous regulatory precedent, where phase III osteoporosis trials typically required fracture incidence as the primary endpoint, often extending trial timelines and raising cost and ethical hurdles. By accepting BMD as the primary endpoint, the FDA opens the door for a more streamlined path to innovation in osteoporosis drug development.
“Osteoporosis afflicts more women than heart attack, stroke, and breast cancer combined,” added Toledano. “With over 200 million women affected globally and few treatment innovations in recent years, EB613—our first-in-class oral, once-daily anabolic tablet—has the potential to address a significant unmet need by increasing skeletal mass, improving bone microarchitecture, and reducing fracture risk.”
