March 30, 2026 -- Sobi® (STO: SOBI) today announced that the European Medicines Agency (EMA) has validated an indication extension application for Tryngolza® (olezarsen) for the treatment of adult patients with severe hypertriglyceridemia (sHTG) ≥880 mg/dL (≥10 mmol/L). Patients with elevated triglyceride levels have substantially higher risks of all-cause mortality, atherosclerotic cardiovascular events, and acute pancreatitis[1]. The submission is supported by results from the pivotal Phase 3

Poster #76954 highlights QTORIN™ rapamycin’s single phase anhydrous gel formulation designed to optimize dermal bioavailability of rapamycin for mTOR-driven skin diseases while overcoming the crystallization, stability, and dermal penetration challenges posed by rapamycin Poster #76929 highlights a qualitative patient and caregiver interview study evaluating the burden of living with porokeratosis, a serious, rare genetic skin disease characterized by numerous pre-cancerous, pruritic lesions and

In the ADlong Phase 3b study, nearly all EBGLYSS-treated patients achieved meaningful skin improvement (EASI-75) for up to four years 75% of patients achieved a high bar of near-complete skin clearance (EASI-90) and 78% experienced significant itch relief (Pruritus NRS ≤4), one of the most bothersome symptoms for patients 80% of patients achieved durable results without the need for topical corticosteroids March 27, 2026 -- New long-term data show Eli Lilly and Company's (NYSE: LLY) EBGLYSS (le

Long-term subcutaneous SPEVIGO ® (spesolimab-sbzo) treatment reduced flare frequency in patients with generalized pustular psoriasis (GPP), with nearly 75% experiencing no flares over three years. 1 Rapid flare control was observed, with 50% of patients achieving a GPPGA pustulation subscore of 0 at Week 1 with intravenous SPEVIGO. 2 LEO Pharma A/S, a global leader in medical dermatology, today announced new long-term results from EFFISAYIL® ON, an ongoing five-year open-label extension study o

- Improvements seen across all six DLQI Domains indicating broad, beneficial impact on quality of life - - Data further demonstrates first-in-class and best-in-disease barzolvolimab profile - March 27, 2026 -- Celldex (NASDAQ:CLDX) presented additional positive data from the completed Phase 2 clinical trials of barzolvolimab in chronic spontaneous urticaria (CSU) and cold urticaria (ColdU) and symptomatic dermographism (SD) demonstrating profound improvements in patient quality of life (QoL) a

First-line ZEGFROVY® monotherapy demonstrated an objective response rate (ORR) of 81.3% in patients with advanced NSCLC harboring EGFR PACC or other uncommon mutations, with a manageable safety profile March 27, 2026 -- Dizal (SSE:688192), a biopharmaceutical company committed to developing novel medicines for cancer and immunological diseases, today announced the presentation of new clinical data for ZEGFROVY® (sunvozertinib) as a first-line treatment in patients with advanced non-small cell l

The US FDA has granted accelerated approval to Kresladi (marnetegragene autotemcel; RP-L201), an autologous hematopoietic stem cell-based gene therapy developed by Rocket Pharmaceuticals for pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) caused by biallelic variants in the ITGB2 gene. The approval makes Kresladi the first gene therapy cleared by the FDA for this ultra-rare primary immunodeficiency. The agency also granted Rocket a Rare Pediatric Disease Priority Review Vo

FDA grants Orphan Drug Designation to SignaBlok's first-in-class, new mechanism-based TREM-1 peptide inhibitor to treat retinopathy of prematurity March 26, 2026 -- SignaBlok, Inc., a preclinical stage biotechnology company pioneering novel, first-in-class peptide therapies for multiple inflammation-associated diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to the Company’s TREM-1 peptide inhibitor for the treatment of retinopathy of

While not reaching statistical significance, there was a slowing of disease progression relative to placebo, as measured by IBMFRS, in the overall patient population at the lower dose of ulviprubart, with a 50% slowing of disease progression relative to placebo across both doses in a pre-specified subgroup of patients with less severe disease Ulviprubart demonstrated a favorable safety and tolerability pro with placebo in the study Study validates ulviprubart’s mechanism of targeted depletion

Beam Therapeutics reported updated clinical data from its ongoing Phase I/II trial of BEAM-302, a lipid-nanoparticle-delivered base editing therapy under development for alpha-1 antitrypsin deficiency. According to the firm’s press release, a single 60 mg dose raised levels of the protective protein alpha-1 antitrypsin (AAT) above the threshold associated with lung protection in all patients. Mean levels reached 16.1 µM and were sustained across five to twelve months of follow-up, supporting sel