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FDA Authorizes Emergency Compassionate Use of VAR 200 for Ultra-Rare ApoCII Amyloidosis

Rare DiseasePrecision MedicineOrphan Drug
  • The FDA has authorized emergency compassionate use of ZyVersa Therapeutics' VAR 200 for a patient with ApoCII amyloidosis, an ultra-rare kidney disease with no effective treatment options.
  • VAR 200, currently in Phase 2a trials for diabetic kidney disease, targets lipid accumulation in kidneys and was selected based on evidence of lipotoxic glomerular injury in the patient's kidney biopsy.
  • The treatment will be administered at the University of Miami under Dr. Alessia Fornoni's care, following the protocol of the ongoing diabetic kidney disease clinical trial.
  • This compassionate use case represents a precision medicine approach to match patients with specific molecular signatures to targeted therapies for rare diseases.

P2a Open Label Study to Evaluate 2-HPβCD in Subjects With Diabetic Kidney Disease

NCT06489340RecruitingPhase 2
ZyVersa Therapeutics, Inc.
Posted 6/11/2025

Basilea Secures $39 Million BARDA Funding to Advance Novel Antifungal Candidates Fosmanogepix and BAL2062

Orphan Drug
  • Basilea Pharmaceutica received $39 million in additional BARDA funding to continue development of novel antifungals fosmanogepix and BAL2062, bringing total committed funding to $68 million under a potential $268 million agreement.
  • The funding will support ongoing Phase 3 trials for fosmanogepix in yeast infections and an upcoming Phase 3 study in mold infections, plus preparation for Phase 2 trials of BAL2062.
  • Both antifungal candidates feature novel mechanisms of action and target life-threatening infections in immunocompromised patients, addressing critical unmet medical needs in treating drug-resistant fungal infections.
  • Fosmanogepix has demonstrated activity against multi-drug-resistant strains including Candida auris and rare difficult-to-treat molds, while BAL2062 shows fungicidal activity against azole-resistant Aspergillus species.

A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Patients With Invasive Mold Infections.

NCT06925321RecruitingPhase 3
Basilea Pharmaceutica
Posted 8/26/2025

Study to Assess Safety, Tolerability and Pharmacokinetics of APX001 Administered by Intravenous Infusion to Healthy Subjects

NCT02956499CompletedPhase 1
Basilea Pharmaceutica
Posted 5/24/2016

A Phase 3 Efficacy and Safety Study of Fosmanogepix for the Treatment of Adult Participants With Candidemia and/or Invasive Candidiasis.

NCT05421858RecruitingPhase 3
Basilea Pharmaceutica
Posted 12/11/2024

Neurocrine Biosciences Advances CAH Treatment Portfolio with Long-Acting Injectable Therapy

Rare DiseaseOrphan Drug
  • Neurocrine Biosciences initiated Phase 1 trials for NBIP-01435, a long-acting injectable CRF1 receptor antagonist for congenital adrenal hyperplasia (CAH), complementing its recently approved oral therapy CRENESSITY.
  • CRENESSITY demonstrated significant clinical benefits in Phase 3 trials, achieving 27% glucocorticoid dose reduction in adults and 18% in pediatric patients while maintaining hormonal control.
  • The dual-mechanism approach positions Neurocrine to capture the majority of the CAH market, projected to exceed $1.5 billion by 2030, with regulatory protections including seven years of market exclusivity.
  • Q1 2025 sales of CRENESSITY reached $14.5 million with 413 patient starts, while analysts project $500 million in annual sales by 2027.

MeiraGTx Advances Gene Therapy Pipeline with FDA RMAT Designation and Pivotal Trial Progress

Orphan Drug
  • MeiraGTx received FDA RMAT designation for AAV-GAD in Parkinson's disease and aligned with the FDA on its Phase 2 AQUAx2 study for radiation-induced xerostomia to support potential BLA filing.
  • The company demonstrated significant disease-modifying effects in Parkinson's disease through FDG-PET imaging data showing changes in pathological basal ganglia circuitry, including the substantia nigra.
  • MeiraGTx is preparing to file for regulatory approval of AAV-AIPL1 for LCA4 in Q4 2025, with all 11 treated children showing meaningful responses and those blind at birth now able to see.
  • The company secured $200 million in funding through its strategic collaboration with Hologen AI to advance AAV-GAD development and other CNS therapies.

A Study of AAV2-hAQP1 Gene Therapy in Participants With Radiation-Induced Late Xerostomia

NCT05926765RecruitingPhase 2
MeiraGTx, LLC
Posted 6/13/2023

FELIQS Secures $9 Million Series A to Advance Novel ROP Prevention Therapy FLQ-101

Orphan Drug
  • FELIQS Corporation completed a $9 million Series A funding round co-led by a major American pharmaceutical company and Beyond Next Ventures to advance FLQ-101 for retinopathy of prematurity prevention.
  • FLQ-101 is a first-in-class small molecule targeting oxidized lipids that can be administered once daily orally or intravenously to enhance physiological retinal vascularization.
  • The FDA has granted FLQ-101 both Fast Track and Orphan Drug designations, with the Phase 1b/2 tROPhy-1 study scheduled to begin in summer 2025.
  • The funding will support expansion of operational and clinical development teams while accelerating key milestones in the U.S. clinical program for this underserved pediatric population.

Soligenix Completes Manufacturing Transfer of Synthetic Hypericin for Rare Skin Cancer Treatment

Orphan DrugRare Disease
  • Soligenix successfully transferred manufacturing of synthetic hypericin from Europe to the U.S. through partnership with Sterling Pharma Solutions, establishing scalable cGMP production for clinical trials.
  • The synthetic hypericin serves as the active ingredient in HyBryte™ and SGX302, topical photodynamic therapies being developed for cutaneous T-cell lymphoma and psoriasis treatment.
  • HyBryte™ demonstrated significant efficacy in Phase 3 trials with 16% of patients achieving 50% lesion reduction versus 4% with placebo, leading to FDA orphan drug and fast track designations.
  • A second confirmatory Phase 3 trial (FLASH2) is planned to enroll approximately 80 patients with early-stage CTCL, following EMA agreement on key design components.

IntraBio Completes Phase III Recruitment for Ataxia-Telangiectasia Treatment in Record Time

Rare DiseaseOrphan Drug
  • IntraBio's Phase III pivotal trial for N-Acetyl-L-Leucine (IB1001) in Ataxia-Telangiectasia completed recruitment in under two months, over-enrolling by 167%.
  • The randomized, placebo-controlled, double-blind crossover study enrolled patients across ten multinational sites in six countries.
  • Data readout is expected in Q1 2026, potentially bringing the first approved treatment for A-T patients closer to reality.
  • The rapid enrollment demonstrates significant unmet medical need and community enthusiasm for this investigational neurological therapy.

A study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia

2024-517706-29-00Active, Not RecruitingPhase 3
Intrabio Limited
Posted 3/18/2025

Genenta Reports 3-Year Survival in Glioblastoma Patients with Temferon Cell Therapy

Orphan Drug
  • Two patients with newly diagnosed glioblastoma multiforme have survived three years from initial surgery in Genenta's TEM-GBM trial, with one showing no disease progression and the other achieving disease stabilization without additional therapy.
  • The phase 1/2a trial demonstrated a 29% two-year survival rate and 17-month median overall survival in unmethylated MGMT patients, compared to historical rates of 14% and 13-15 months respectively.
  • Temferon represents a first-in-class hematopoietic stem cell therapy that reprograms the tumor microenvironment by delivering interferon-alpha directly to tumors through engineered myeloid cells.
  • Genenta has initiated the TEM-GU phase 1 study to evaluate Temferon in genitourinary cancers, with plans to demonstrate safety in metastatic renal cell carcinoma by year-end.

A Study Evaluating Temferon in Patients with Glioblastoma & Unmethylated MGMT

NCT03866109RecruitingPhase 1
Genenta Science
Posted 3/5/2019

A Clinical Gene Therapy Study with Hematopoietic Stem Cells for the Treatment, with Single Dose of Temferon, of Patients Suffering from Metastatic Renal Cell Carcinoma

NCT06716853RecruitingPhase 1
Genenta Science
Posted 10/22/2024

Schrödinger's SGR-1505 Receives FDA Fast Track Designation for Relapsed Waldenström Macroglobulinemia

Orphan Drug
  • Schrödinger's MALT1 inhibitor SGR-1505 received FDA Fast Track designation for treating adult patients with Waldenström macroglobulinemia who have failed at least two lines of therapy, including a BTK inhibitor.
  • The designation addresses significant unmet medical need as treatment failure and disease progression due to BTK resistance remains a challenge for a growing number of patients with hematologic malignancies.
  • Phase 1 clinical data presented at recent conferences showed SGR-1505 has a favorable safety profile and encouraging preliminary efficacy across multiple B-cell malignancy subtypes.
  • The company plans to discuss Phase 1 study results and recommended Phase 2 dose with the FDA later this year, with potential for accelerated approval pathways.

Study of SGR-1505 in Mature B-Cell Neoplasms

NCT05544019RecruitingPhase 1
Schrödinger, Inc.
Posted 4/10/2023

First European Patient Receives Breakthrough Treatment for Ultra-Rare APDS Immune Disorder

Rare DiseaseTargeted TherapyDrug ApprovalOrphan Drug
  • Mary Catchpole, 19, becomes the first European patient to receive leniolisib (Joenja), a newly approved targeted treatment for activated PI3-kinase delta syndrome (APDS), a rare inherited immune disorder.
  • The drug works by inhibiting an overactive enzyme that disrupts immune function, offering a potential cure for patients who previously faced lifelong infections and invasive treatments.
  • APDS was discovered by Cambridge researchers in 2013 with help from Catchpole's family, who lost four members to the condition before this breakthrough treatment became available.
  • The NHS approved leniolisib at a list price of £352,000 per year with a confidential discount, potentially benefiting up to 50 patients over age 12 in England.

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