FDA Approves Sobi’s GAMIFANT as the First Treatment for Macrophage Activation Syndrome in Still’s Disease 

In a major advancement for rare inflammatory conditions, the FDA has approved GAMIFANT (emapalumab-lzsg) for the treatment of hemophagocytic lymphohistiocytosis (HLH)/macrophage activation syndrome (MAS) in patients with known or suspected Still’s disease, including systemic juvenile idiopathic arthritis (sJIA) and adult-onset Still’s disease. The approval spans both adult and pediatric populations, including newborns, who show inadequate response or intolerance to glucocorticoids or experience recurrent MAS episodes.

“This approval brings much-needed hope to a vulnerable patient population. GAMIFANT now offers a targeted therapy for MAS in Still’s disease, something we previously lacked,” said Dr. Alexei A. Grom, Professor of Pediatrics at Cincinnati Children’s Hospital. “It allows us to better control hyperinflammation and reduce reliance on high-dose steroids.”

The FDA’s decision was supported by pooled data from the pivotal Phase III EMERALD study (NCT05001737) and NI-0501-06 (NCT03311854). Results showed that 54% of patients achieved complete response at Week 8, while 82% achieved clinical remission (VAS ≤1 cm). The safety profile remained consistent with prior trials, with viral infections and rash being the most common adverse events.

“With our expertise in HLH, we understand the urgency of managing MAS quickly to improve outcomes,” said Guido Oelkers, CEO of Sobi. “GAMIFANT has already proven its value in primary HLH, and we are proud to extend its benefits to those battling MAS in Still’s disease.”

GAMIFANT, a monoclonal antibody that blocks interferon gamma (IFNγ), is now the first and only FDA-approved treatment for HLH/MAS in Still’s disease. The condition is characterized by life-threatening hyperinflammation, persistent fever, cytopenias, coagulopathies, and multi-organ complications, highlighting the urgent need for targeted treatment options.

GSK Slashes Price of RTS,S Malaria Vaccine for Children in Endemic Countries to Under $5

In a significant move toward accessible malaria prevention, Bharat Biotech and GSK have announced a dramatic price reduction for the RTS,S malaria vaccine, lowering its cost to below $5 per dose by 2028. This commitment, made as part of pledges to Gavi’s 2026–2030 replenishment phase (Gavi 6.0), is the result of expanded production, efficient manufacturing, and minimal profit margins, aimed at improving vaccine affordability and accessibility in endemic African nations.

Dr. Krishna Ella, Executive Chairman of Bharat Biotech, emphasized the broader impact of this move: “This pledge to Gavi 6.0 is more than a pricing announcement. It’s a commitment to global equity, innovation, and collaboration. Together with GSK and our global partners, we aim to transform the malaria burden for millions of children and families.”

Developed by GSK, PATH, and partners, RTS,S was the first malaria vaccine recommended by the WHO in 2021. Since then, GSK has invested heavily in scaling production and transferring technology to Bharat Biotech, which in turn has dedicated over $200 million toward new high-output facilities and development. The partnership has enabled a phased price reduction, with full transition of production to Bharat Biotech expected by 2028.

“We partnered with Bharat Biotech in 2021 with a shared goal to get ahead of malaria,” said Thomas Breuer, Chief Global Health Officer at GSK. “This long-term price ambition demonstrates our joint commitment to infectious disease control and public health impact, especially for children across Africa.”

With support from Gavi, the RTS,S vaccine is expected to be integrated into routine immunisation programs across 12 African countries by the end of 2025. A recent WHO evaluation of RTS,S implementation in Ghana, Kenya, and Malawi (2019–2023) showed a 13% reduction in all-cause mortality and a 22% drop in severe malaria hospitalizations among vaccinated children. These numbers highlight the vaccine’s real-world impact in high-transmission regions.

Dr. Sania Nishtar, CEO of Gavi, noted, “We are grateful to GSK and Bharat Biotech for their innovation and commitment. This partnership enables us to protect millions more children across Africa—bringing our 2030 goal of reaching 50 million children within reach.”

Further research is already underway at GSK to develop next-generation malaria vaccines, targeting different stages of the parasite’s lifecycle, to expand the malaria prevention toolkit and advance global eradication efforts.

Schrödinger Receives FDA Fast Track Designation for SGR-1505 in Relapsed/Refractory Waldenström Macroglobulinemia

Schrödinger, Inc. has announced that its investigational drug SGR-1505, a clinical-stage MALT1 inhibitor, has been granted Fast Track designation by the FDA for the treatment of adult patients with relapsed/refractory Waldenström macroglobulinemia who have previously failed at least two lines of therapy, including a BTK inhibitor.

“We are excited to receive Fast Track designation for SGR-1505, which underscores the significant need in patients with Waldenström macroglobulinemia,” said Dr. Karen Akinsanya, President and Head of Therapeutics R&D at Schrödinger. “BTK resistance remains a growing challenge, and SGR-1505’s novel MALT1 mechanism offers potential as both monotherapy and part of combination regimens.”

The Fast Track status is designed to accelerate the development and review process for drugs addressing serious medical conditions and unmet needs, potentially offering benefits such as more frequent interactions with the FDA, rolling submissions, and eligibility for Priority Review and Accelerated Approval.

SGR-1505 is currently under evaluation in a Phase I clinical trial for relapsed/refractory B-cell malignancies, including chronic lymphocytic leukemia (CLL), diffuse large B-cell lymphoma (DLBCL), and marginal zone lymphoma (MZL). Initial results, recently presented at major hematology congresses, demonstrated a favorable safety profile and preliminary signs of efficacy, including in BTK-pretreated Waldenström patients.

“The Fast Track designation, along with our Phase I data across several B-cell malignancies, highlights SGR-1505’s potential as a meaningful therapeutic option,” said Dr. Margaret Dugan, Chief Medical Officer at Schrödinger. “We look forward to engaging with the FDA later this year to discuss the next phase of development.”

SGR-1505 was also granted Orphan Drug Designation by the FDA in August 2023 for Mantle Cell Lymphoma (MCL), further supporting its development in B-cell cancers with high unmet need.

Ultragenyx Granted Breakthrough Therapy Designation by FDA for GTX-102 in Angelman Syndrome

Ultragenyx Pharmaceutical Inc. announced that the FDA has granted Breakthrough Therapy Designation to GTX-102 (apazunersen) for the treatment of Angelman syndrome, a rare neurogenetic disorder with no approved therapies to date.

“FDA Breakthrough Therapy Designation underscores both the urgent need for an effective treatment for patients and families affected by Angelman syndrome and the clinically meaningful results demonstrated to date with GTX-102,” said Eric Crombez, M.D., Chief Medical Officer at Ultragenyx. “With strong support from the Angelman community, our Phase III Aspire study is enrolling rapidly, and we’re committed to moving GTX-102 through development as quickly as possible.”

The designation is based on promising clinical data from a Phase I/II study involving 74 pediatric patients (aged 4–17) with a full maternal UBE3A gene deletion, showing rapid, sustained, and ongoing improvements across multiple developmental domains after up to three years of treatment. These early results suggest substantial benefit over existing therapeutic options and support the drug’s potential as a disease-modifying therapy.

The Phase III Aspire study (NCT06617429) began enrollment in December 2024 and is expected to recruit approximately 120 children with genetically confirmed Angelman syndrome. In addition, Ultragenyx plans to launch the Aurora study in the second half of 2025 to explore GTX-102 in other genotypes and age groups, expanding the therapy’s potential reach.

GTX-102 continues to generate strong interest within the rare disease community, and its Breakthrough Therapy Designation marks a significant regulatory milestone in addressing a long-standing unmet need in Angelman syndrome.

FDA Approves Updated Label for Eli Lilly’s AMYVID to Aid Alzheimer’s Disease Diagnosis

Eli Lilly and Company announced that the FDA has approved a label update for AMYVID (florbetapir F 18 injection), a diagnostic imaging agent used to estimate amyloid plaque density in the brains of patients being evaluated for Alzheimer’s disease and related cognitive disorders.

The updated label expands AMYVID’s use to aid in selecting patients for amyloid-targeting therapies, offering more flexibility in diagnosing and managing Alzheimer’s-related conditions. It also allows quantitative assessment of amyloid plaque levels, in addition to traditional visual interpretation. The revised label also reflects AMYVID’s role in evaluating treatment response in clinical trials of amyloid-targeting drugs.

“The modernization of AMYVID’s label is a significant step in advancing Alzheimer’s care,” said Mark Mintun, M.D., Group Vice President, Neuroscience R&D at Lilly. “It enables physicians to better support patients by helping them make more informed decisions, especially those being considered for amyloid-targeting therapies.”

AMYVID binds to amyloid plaques, abnormal protein clumps associated with Alzheimer’s, and makes them visible via positron emission tomography (PET) imaging. A positive scan indicates a higher likelihood of Alzheimer’s disease, while a negative scan suggests minimal amyloid buildup, making Alzheimer’s less likely.

The update comes at a time when early and accurate diagnosis of Alzheimer’s is becoming increasingly critical. According to Lilly, nearly 80% of Americans prefer knowing their Alzheimer’s status before significant cognitive decline, reinforcing the need for advanced diagnostics such as amyloid PET scans and blood biomarker testing.

AMYVID is widely reimbursed under Medicare and private insurance plans, although coverage may vary. The updated label aims to improve diagnostic accuracy, guide therapy decisions, and ultimately enhance patient outcomes in the evolving Alzheimer’s treatment landscape.