March 30, 2026 -- Nanoscope Therapeutics, Inc., a biotechnology company committed to developing and commercializing novel, disease-agnostic therapies for patients with photoreceptor loss and vision impairment due to retinal degeneration, today announced that it has received Orphan Drug Designation in the Kingdom of Saudi Arabia for its lead therapy, MCO-010, for the treatment of patients with inherited retinal dystrophies (IRDs) having severe vision loss. The broad designation from Saudi regula

KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) today announced new interim results from its KONFIDENT-KID clinical trial evaluating EKTERLY® (sebetralstat) for the on-demand treatment of hereditary angioedema (HAE) attacks in children ages 2-11 presented at the 2026 Global Angioedema Leadership Conference. KONFIDENT-KID is the largest pediatric trial ever conducted in HAE. It features a proprietary oral disintegrating tablet (ODT) formulation of sebetralstat in a population that currently relies

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