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RemeGen's Telitacicept Receives Approval in China for Generalized Myasthenia Gravis Treatment

Drug ApprovalRare Disease
  • RemeGen announced that Telitacicept has officially been approved for marketing in China by the National Medical Products Administration for treating generalized myasthenia gravis.
  • The approval represents a significant milestone for patients with this rare autoimmune neuromuscular disorder in China.
  • This regulatory decision expands the therapeutic options available for generalized myasthenia gravis patients in the Chinese market.

SKYTROFA Receives Australian Approval as First Once-Weekly Growth Hormone Therapy for Children

Rare DiseaseDrug Approval
  • The Therapeutic Goods Administration has approved SKYTROFA (lonapegsomatropin) as a once-weekly injectable treatment for pediatric growth hormone deficiency in children aged 3-18 years.
  • The therapy addresses a rare condition affecting approximately 2,000 Australian children, offering a significant improvement over daily injection regimens previously required.
  • SKYTROFA utilizes Ascendis Pharma's TransCon technology platform to provide sustained release of unmodified growth hormone over one week.
  • The approval was based on three pivotal Phase 3 clinical trials that collectively treated over 300 pediatric patients with growth hormone deficiency.

A Trial to Compare the Efficacy and Safety of Once-weekly Lonapegsomatropin With Placebo and a Daily Somatropin Product in Adults With Growth Hormone Deficiency

NCT04615273CompletedPhase 3
Ascendis Pharma Endocrinology Division A/S
Posted 12/3/2020

Lantern Pharma's LP-184 Demonstrates Significant Survival Benefits in Pediatric Brain Cancer Models

Rare DiseaseOrphan Drug
  • LP-184 showed remarkable survival improvements in mouse models of atypical teratoid rhabdoid tumors (ATRT), with median survival increasing by 345% in one model (p<0.0001).
  • Independent research from Johns Hopkins University validates Lantern Pharma's FDA Rare Pediatric Disease Designation for LP-184 in treating this aggressive pediatric brain cancer.
  • The drug demonstrated potent anti-tumor activity across multiple ATRT cell lines with strong blood-brain barrier penetrance and no apparent toxicity in preclinical studies.
  • Lantern Pharma plans to initiate a pediatric Phase I clinical trial for LP-184 in brain tumors in late 2025 or early 2026.

Study of LP-184 in Patients with Advanced Solid Tumors

NCT05933265RecruitingPhase 1
Lantern Pharma Inc.
Posted 6/9/2023

uniQure's Gene Therapy AMT-260 Achieves 92% Seizure Reduction in First Drug-Resistant Epilepsy Patient

Rare Disease
  • uniQure's investigational gene therapy AMT-260 demonstrated a 92% reduction in seizure frequency in the first patient treated for drug-resistant mesial temporal lobe epilepsy during five months of follow-up.
  • The patient experienced no serious adverse events and went seizure-free for the final 60 days of the monitoring period, showing promising early safety and efficacy signals.
  • AMT-260 works by delivering engineered microRNAs to suppress the GRIK2 gene and aberrant GluK2 expression, targeting a key mechanism believed to trigger seizures in refractory MTLE.
  • The GenTLE Phase I/IIa trial is actively enrolling patients across 12 U.S. sites, addressing a significant unmet need for over 600,000 Americans with temporal lobe epilepsy.

AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy

NCT06063850RecruitingPhase 1
UniQure Biopharma B.V.
Posted 6/12/2024

Drug Farm's DF-003 Shows Promising Safety Profile in Phase 1 Trial for Rare ROSAH Syndrome

Rare DiseasePrecision Medicine
  • Drug Farm reported positive Phase 1 results for DF-003, a first-in-class ALPK1 inhibitor targeting ROSAH syndrome, showing excellent safety with no serious adverse events across all tested doses.
  • The randomized, placebo-controlled study in 48 healthy volunteers demonstrated dose-proportional pharmacokinetics supporting once-daily oral administration for future trials.
  • DF-003 achieved blood concentrations consistent with efficacy in preclinical models, positioning the company to advance into proof-of-concept trials for ROSAH syndrome and cardio-renal disease.
  • The drug represents a potential breakthrough for ROSAH syndrome, a rare genetic disease with no approved treatments that causes progressive vision loss and inflammatory symptoms.

Evaluating the Safety, Tolerability, and Pharmacokinetics of DF-003 in Healthy Subjects

NCT05997641Active, Not RecruitingPhase 1
Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Posted 9/15/2023

Evaluating the Safety and Tolerability of Orally Administered DF-003 in ROSAH Syndrome Patients

NCT06395285RecruitingPhase 1
Shanghai Yao Yuan Biotechnology Ltd. (also known as Drug Farm)
Posted 4/1/2025

Eton Pharmaceuticals Receives FDA Approval for KHINDIVI, First Hydrocortisone Oral Solution for Pediatric Adrenal Insufficiency

Drug ApprovalRare Disease
  • Eton Pharmaceuticals announced FDA approval of KHINDIVI (hydrocortisone) oral solution, marking the first and only FDA-approved liquid hydrocortisone formulation for pediatric patients aged 5 and older with adrenocortical insufficiency.
  • The ready-to-use 1mg/ml oral solution eliminates the need to split or crush tablets, offering precise dosing for children who have difficulty swallowing pills or require gastric tube administration.
  • Commercial launch is expected the week of June 2nd, with Eton projecting combined peak sales of KHINDIVI and ALKINDI SPRINKLE to exceed $50 million annually.
  • The approval addresses a decades-long unmet need in pediatric endocrinology, providing accurate dosing capabilities critical for managing the more than 5,000 U.S. patients aged 5-17 with adrenal insufficiency.

DEBRA Research and Dermaliq Partner to Develop Touchless Drug Delivery for Epidermolysis Bullosa

Rare Disease
  • DEBRA Research and Dermaliq Therapeutics announced a strategic collaboration to advance cutaneous drug delivery for epidermolysis bullosa using Dermaliq's proprietary hyliQ® touchless technology platform.
  • The partnership will initially focus on developing innovative topical treatments for wound healing and itch relief, plus prophylactic therapies for people with epidermolysis bullosa.
  • Dermaliq's hyliQ® technology enables targeted delivery of active compounds into fragile skin without mechanical interference, specifically designed for conditions where skin is exceptionally delicate.
  • DEBRA Research has made a strategic investment in Dermaliq as part of the non-exclusive collaboration agreement.

Alvotech and Advanz Pharma Expand $13.8 Billion Biosimilar Partnership with Three New Candidates

Monoclonal AntibodyRare Disease
  • Alvotech and Advanz Pharma have expanded their strategic partnership to include three additional biosimilar candidates, bringing their total European portfolio to more than ten reference products.
  • The new agreement covers biosimilars to Ilaris (canakinumab) for inflammatory diseases and Kesimpta (ofatumumab) for multiple sclerosis, plus an undisclosed early-stage candidate.
  • The partnership includes development and commercial milestones totaling up to $180 million, with the companies participating in revenue sharing arrangements.
  • The expanded collaboration increases the addressable market for their combined biosimilar portfolio to at least $13.8 billion across European territories.

Pimicotinib Achieves 54% Response Rate in Phase 3 Trial for Rare Joint Tumor

Rare DiseaseOncology
  • Pimicotinib demonstrated a statistically significant 54.0% objective response rate versus 3.2% for placebo in the global Phase 3 MANEUVER trial for tenosynovial giant cell tumor.
  • The CSF-1R inhibitor met all five secondary endpoints, showing clinically meaningful improvements in pain, stiffness, range of motion, and physical function.
  • Nearly two-thirds of patients experienced at least 50% reduction in tumor volume, with treatment well-tolerated and low discontinuation rates.
  • Results represent the highest response rate achieved to date with systemic therapy for TGCT, with regulatory submissions planned to begin this year.

Study of Pimicotinib (ABSK021) for Tenosynovial Giant Cell Tumor (MANEUVER)

NCT05804045Active, Not RecruitingPhase 3
Abbisko Therapeutics Co, Ltd
Posted 4/27/2023

Sobi to Present Clinical Data on Rare Disease Therapies at EULAR 2025

Rare DiseaseMonoclonal Antibody
  • Swedish Orphan Biovitrum (Sobi) will present new clinical data at EULAR 2025 in Barcelona, including efficacy and safety outcomes for Gamifant in treating macrophage activation syndrome.
  • The company will share updates on Vonjo's potential treatment of VEXAS syndrome and analysis on uncontrolled gout management approaches.
  • Sobi will host a dedicated symposium on VEXAS syndrome's dermatologic, rheumatologic, and hematologic features, chaired by Dr. Sophie Georgin-Lavialle from the French National Reference Centre.
  • The presentations will showcase multiple investigational therapies targeting rare inflammatory and hematologic conditions with significant unmet medical needs.

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