Regeneron’s EVKEEZA Wins FDA Approval for HoFH in Children as Young as 1 Year

Regeneron Pharmaceuticals, Inc. announced that the FDA has approved EVKEEZA (evinacumab-dgnb) ANGPTL3 antibody, for children aged 1 to under 5 years with homozygous familial hypercholesterolemia (HoFH). This expands the indication from its previous approval for children aged 5–11 and adolescents and adults aged 12 and older. EVKEEZA is approved as an adjunct to diet, exercise, and other lipid-lowering therapies.

“The approval of EVKEEZA for children as young as 1 year addresses a critical unmet need for those with HoFH, a life-threatening condition causing extremely high LDL-C levels from birth,” said Katherine Wilemon, Founder and CEO of the Family Heart Foundation. “Families and their medical teams now have an effective treatment option for these very young patients at risk of serious cardiovascular complications.”

HoFH is a rare genetic disorder affecting approximately 1,300 people in the U.S., resulting in LDL-C levels often exceeding 400 mg/dL. Patients are at risk of premature atherosclerotic disease and cardiac events, sometimes during their teenage years. EVKEEZA’s initial approval in 2021 was based on a placebo-controlled trial showing about 50% LDL-C reduction when added to standard lipid-lowering therapies. The extended indication was supported by clinical efficacy and safety data from 6 children treated under expanded access or compassionate use programs, with no new safety concerns identified.

“EVKEEZA is a testament to the power of Regeneron’s science and proprietary technologies in developing first-in-class, life-changing medicines,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and CSO at Regeneron. “This label extension reinforces our commitment to rare diseases, including neuromuscular and genetic disorders, where we continue to advance innovative therapies.”

FDA Approves Eli Lilly’s INLURIYO for ER+, HER2-, ESR1-Mutated Advanced Breast Cancer

Eli Lilly and Company announced that the FDA has approved INLURIYO (imlunestrant, 200 mg tablets) for adults with estrogen receptor-positive (ER+), HER2-negative, ESR1-mutated advanced or metastatic breast cancer (MBC) whose disease progressed after at least one line of endocrine therapy. INLURIYO is a once-daily oral estrogen receptor antagonist designed to block and degrade overactive estrogen receptors, slowing tumor progression.

“This therapy reflects our commitment to developing treatments that improve outcomes for people with breast cancer and represents an important step toward advancing innovative, all-oral treatment approaches,” said Jacob Van Naarden, EVP and President of Lilly Oncology. “We are deeply grateful to the patients, investigators, Lilly team members, and clinical care teams who made this advancement possible.”

The FDA approval is based on the Phase III EMBER-3 trial, in which INLURIYO reduced the risk of progression or death by 38% versus standard endocrine therapy in patients with ESR1-mutated MBC. Median progression-free survival was 5.5 months versus 3.8 months for fulvestrant or exemestane (HR=0.62, p=0.0008). Adverse events were mostly low grade, with 4.6% of patients discontinuing due to side effects.

“This represents an important advancement for patients with ESR1-mutated MBC, a mutation found in nearly half of those previously treated with hormone therapies,” said Komal Jhaveri, M.D., FACP, FASCO, Memorial Sloan Kettering Cancer Center. “With its efficacy, tolerability, and oral administration, INLURIYO provides a meaningful alternative treatment option for this patient population.”

Janssen’s TREMFYA Secures FDA Nod for Pediatric Plaque Psoriasis and Psoriatic Arthritis

Johnson & Johnson announced that the FDA has approved TREMFYA (guselkumab) for children aged six years and older, weighing at least 40 kg, with moderate to severe plaque psoriasis (PsO) or active psoriatic arthritis (PsA). This milestone makes TREMFYA the first and only IL-23 inhibitor approved for these pediatric indications, expanding upon its earlier adult approvals for PsO in 2017 and PsA in 2020.

“Despite advancements in treatment, there continues to be a significant gap in therapies for children living with these debilitating immune-mediated diseases,” said Vimal Hasmukh Prajapati, M.D., University of Calgary and Co-Director of the Skin Health & Wellness Centre. “TREMFYA offers physicians and families an established option with proven safety and efficacy to improve symptoms and quality of life.”

The plaque PsO approval was supported by the Phase III PROTOSTAR study, where TREMFYA achieved co-primary endpoints of PASI 90 and IGA 0/1 at Week 16. Approximately 56% of patients reached PASI 90 versus 16% on placebo, and nearly 40% achieved complete clearance (IGA 0). The active PsA indication was supported by pharmacokinetic extrapolation from adult PsO and PsA studies, confirming safety and efficacy in pediatric patients.

“Every child deserves to be active without limitations from joint pain or skin symptoms,” said Brandee Pappalardo, PhD, MPH, VP, Medical Affairs, Dermatology & Rheumatology, J&J Innovative Medicine. “The approval of TREMFYA’s pediatric indications represents a meaningful step forward for children and their care partners, reflecting our commitment to chronic immune-mediated disease research.”

TREMFYA is administered subcutaneously at Week 0, Week 4, and every 8 weeks thereafter, with a recommended dose of 100 mg for eligible pediatric patients. This approval underscores Johnson & Johnson’s ongoing dedication to advancing treatments for chronic immune-mediated diseases in both adult and pediatric populations.

Kedrion Biopharma’s QIVIGY (Immune Globulin 10% IV) Approved by FDA for Primary Humoral Immunodeficiency in Adults

Kedrion Biopharma announced that the FDA has approved QIVIGY (immune globulin intravenous [human]-kthm 10% liquid) for adults with primary humoral immunodeficiency (PI), a group of rare disorders that compromise the immune system. QIVIGY, developed and manufactured exclusively by Kedrion in the U.S. and Italy, addresses the unmet needs of patients living with PI and joins Kedrion’s expanding global portfolio of therapies.

“Seeing QIVIGY receive FDA approval is a deeply meaningful moment for all of us at Kedrion,” said Ugo Di Francesco, CEO, Kedrion. “It reflects our scientific capabilities and our mission to serve patients. Today, we celebrate a product that improves patient care while demonstrating our organization’s ability to deliver.”

The FDA approval was supported by a 12-month clinical study in adults with PI, where QIVIGY met its primary endpoint with no acute serious bacterial infections. Secondary outcomes showed a low rate of other infections, no infection-related hospitalizations, and minimal disruption to daily life. 

Common adverse events included headache, infusion reactions, fatigue, and nausea. “For individuals living with PI, fewer infections mean less disruption to daily life, highlighting our commitment to therapies that support patients’ health and well-being,” said Nisha Jain, VP, Global Clinical Development, Kedrion.

QIVIGY represents a strategic milestone for Kedrion, reflecting long-term investments in manufacturing, plasma collection, and R&D to expand rare disease treatments. 

“2025 has been transformative for Kedrion, and QIVIGY marks a pivotal accomplishment as we continue to expand in the U.S. and globally,” said Bob Rossilli, Global Chief Commercial Officer, Kedrion. 

QIVIGY will be launched in the U.S. first, with availability expected in early 2026, and plans for expansion into Europe and other markets upon approval.

FDA Approves Crinetics Pharma’s First Daily Pill, PALSONIFY, for Rare Growth Disorder

The FDA has approved PALSONIFY (paltusotine) from Crinetics Pharmaceuticals for first-line treatment of adults with acromegaly who have an inadequate response to surgery or for whom surgery is not an option. PALSONIFY is the first once-daily, oral treatment for acromegaly, offering a convenient alternative to injectable therapies for this rare disorder, which is characterized by excessive growth hormone and abnormal enlargement of hands, feet, and facial features.

“PALSONIFY represents a major advancement for adults living with acromegaly, providing rapid, sustained biochemical control and meaningful improvements in daily symptoms,” said Joseph Perez, MD, Chief Medical Officer, Crinetics Pharmaceuticals. “We are proud to offer patients an oral therapy that can help improve their quality of life while addressing a critical unmet medical need.”

The approval was based on results from the Phase III PATHFNDR-1 and PATHFNDR-2 trials, which demonstrated PALSONIFY’s consistent efficacy in both previously treated and medically untreated adults. Participants reported significant reductions in signs and symptoms, including headaches, joint pain, sweating, fatigue, and swelling, as measured by the FDA-aligned Acromegaly Symptom Diary (ASD). PALSONIFY was generally well-tolerated, with no serious adverse events reported in the randomized controlled portions of the studies.

“Oral administration of PALSONIFY is a game-changer for patients, reducing the treatment burden while maintaining effective disease control,” said Sarah Thompson, VP of Clinical Development, Crinetics Pharmaceuticals. PALSONIFY is expected to be available in the U.S. in early October. Global expansion plans are underway, including a Marketing Authorization Application under review in the European Union and a partnership with Sanwa Kagaku Kenkyuso (SKK) for development in Japan. The therapy is also being evaluated in Phase III trials for carcinoid syndrome.