Product: AQVESME™

Company: Agios Pharmaceuticals, Inc.

About the Product:

AQVESME™ (mitapivat) is an innovative, oral pyruvate kinase activator designed to improve red blood cell energy and health, protecting against the early breakdown that occurs from conditions like thalassemia – a debilitating disease which has historically had limited or no treatment options. Its U.S. approval is based on results from two global Phase 3 trials demonstrating a significant increase in hemoglobin and reduction in fatigue for non-transfusion-dependent thalassemia patients, as well as a significant reduction in transfusion burden for transfusion-dependent patients. AQVESME is now the only medicine approved for all adults with thalassemia, regardless of their transfusion status or genotype.

Product: JASCAYD® (nerandomilast)

Company: Boehringer Ingelheim

About the Product:

JASCAYD® (nerandomilast) is a preferential phosphodiesterase 4B (PDE4B) inhibitor indicated for the treatment of adults with idiopathic pulmonary fibrosis (IPF) and progressive pulmonary fibrosis (PPF). Building on Boehringer Ingelheim's decades-long legacy in pulmonary fibrosis, JASCAYD represents an important advancement for people living with these serious, progressive lung diseases. Designed to target multiple drivers of disease progression, JASCAYD addresses a significant unmet need and reflects Boehringer's continued commitment to advancing care for patients facing progressive lung scarring, declining lung function, and high mortality.

Product: Attruby® (acoramidis)

Company: BridgeBio Pharma, Inc.

About the Product:

Attruby® (acoramidis) is an oral transthyretin (TTR) stabilizer approved to treat the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults, to reduce cardiovascular death and cardiovascular-related hospitalization. It was rationally designed to mimic a naturally protective genetic variant found in Portuguese families spared from hereditary ATTR amyloidosis, occupying binding sites within the TTR tetramer and holding it together across the dosing interval. Attruby is the only orally administered, selective small-molecule TTR stabilizer to achieve near-complete (≥90%) stabilization, a feature reflected in its U.S. FDA label. This establishes near-complete stabilization as a new standard in a once-untreatable disease.

Product: RYTELO® (imetelstat)

Company: Geron Corporation

About the Product:

RYTELO® (imetelstat) is the first and only FDA-approved telomerase inhibitor, transforming decades of pioneering telomerase research into a first-in-class therapeutic breakthrough for patients with lower-risk myelodysplastic syndromes (LR-MDS) with transfusion-dependent anemia. Rather than primarily managing the consequences of disease, RYTELO targets an underlying biological mechanism by inhibiting telomerase, an enzyme that cancer cells depend on to continue growing and dividing. This novel approach established a new therapeutic approach in LR-MDS while providing a foundation for continued research into the potential of telomerase inhibition across other cancers.

Product: DUVYZAT® (givinostat)

Company: Italfarmaco Group

About the Product:

DUVYZAT® (givinostat) is the first and only nonsteroidal treatment indicated for Duchenne muscular dystrophy (DMD) in people 6 years of age and older, regardless of their genetic variant or ability to walk. An oral, class I/II histone deacetylase (HDAC) inhibitor, DUVYZAT is designed to address downstream disease pathways and the need for a variant-independent approach that targets disease-driving processes that result from HDAC hyperactivity in DMD. This approach allows for treatment of a broad population of patients who historically had limited pharmacologic options beyond corticosteroids.

Product: VIJOICE^®

Company: Novartis

About the Product:

VIJOICE (alpelisib) is the first and only approved treatment for PIK3CA-Related Overgrowth Spectrum (PROS), a rare, progressive disorder previously managed only with repeated surgeries and supportive care. By selectively inhibiting PI3Kα, VIJOICE targets the underlying molecular cause of disease, transforming PROS management from symptomatic intervention to precision medicine. Its development pioneered a novel regulatory pathway, achieving FDA approval based on rigorous real-world evidence from the EPIK-P1 study when conventional trials were not feasible. Beyond delivering meaningful and durable benefits for patients and families, VIJOICE establishes a new framework for precision treatment of rare mosaic genetic disorders.

Product: AVMAPKI^® FAKZYNJA^® CO-PACK

Company: Verastem Oncology

About the Product:

The U.S. Food and Drug Administration (FDA) approved AVMAPKI® FAKZYNJA® CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. AVMAPKI inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors. 

Product: MIPLYFFA^®

Company: Zevra Therapeutics, Inc.

About the Product:

MIPLYFFA®, developed by Zevra Therapeutics, is a first-in-class oral therapy approved for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C, an ultra-rare neurodegenerative disorder affecting about 900 individuals in the U.S. Addressing a critical unmet need, MIPLYFFA halts the progression of disease by penetrating the CNS and targeting disease pathophysiology through a novel mechanism that enhances autophagy and cellular cholesterol clearance. By activating transcription factors TFEB and TFE3, it upregulates the Coordinated Lysosomal Expression and Regulation (CLEAR) gene network, offering a disease-modifying approach that advances the treatment landscape for lysosomal storage disorders.