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FDA Sets Q3 2025 Approval Dates for Three Breakthrough Therapies Targeting Rare Diseases

Drug ApprovalRare DiseaseMonoclonal AntibodyOrphan Drug
  • The FDA has established PDUFA target action dates for three promising therapies in Q3 2025: sepiapterin for phenylketonuria (July 29), apitegromab for spinal muscular atrophy (September 22), and paltusotine for acromegaly (September 25).
  • PTC Therapeutics' sepiapterin demonstrated a 63% reduction in blood phenylalanine levels in PKU patients, with 84% achieving therapeutic control and over 97% able to liberalize their protein-restricted diets.
  • Scholar Rock's apitegromab showed statistically significant motor function improvements in SMA patients already receiving standard care, while Crinetics' paltusotine achieved IGF-1 normalization in 83% of acromegaly patients switching from injectable treatments.
  • These approvals could provide significant therapeutic advances for patients with rare genetic disorders who currently have limited treatment options or face challenges with existing therapies.

Minoryx and Neuraxpharm Submit New Marketing Authorization Application for Leriglitazone in Cerebral Adrenoleukodystrophy

Orphan DrugRare Disease
  • Minoryx Therapeutics and Neuraxpharm have submitted a new Marketing Authorization Application to the EMA for leriglitazone (NEZGLYAL®) to treat cerebral adrenoleukodystrophy, which has been validated and is under review.
  • The application is based on positive results from the NEXUS study, where 35% of pediatric patients met arrested disease criteria compared to the expected 10% natural self-arrest rate.
  • This submission follows a previous regulatory setback in May 2024 when the CHMP recommended against granting marketing authorization after re-examination.
  • If approved, leriglitazone would become the first pharmacological treatment for cALD, a devastating rare disease with no current drug therapy options.

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy

NCT05819866RecruitingPhase 3
Minoryx Therapeutics, S.L.
Posted 7/12/2023

A Clinical Study in Male Pediatric Patients With Cerebral X-linked Adrenoleukodystrophy (Cald) to Assess the Effects of MIN-102 Treatment on Disease Progression Prior to Human Stem Cell Transplant (HSCT)

NCT04528706Active, Not RecruitingPhase 2
Minoryx Therapeutics, S.L.
Posted 9/13/2019

F2G's Novel Antifungal Olorofim Shows Promise Despite FDA Setback, Phase 2b Results Published in The Lancet

Orphan Drug
  • F2G's olorofim, the first in a new class of orotomide antifungals, received a complete response letter from the FDA requiring additional data before approval.
  • Phase 2b study results published in The Lancet Infectious Diseases showed a 28.7% global response rate at day 42 in 202 patients with difficult-to-treat invasive fungal infections.
  • The drug demonstrated efficacy across multiple resistant fungal pathogens including azole-resistant Aspergillus species, with 75.2% of patients achieving successful outcomes when stable disease was included.
  • F2G and partner Shionogi continue development with an ongoing Phase 3 OASIS trial comparing olorofim to standard therapy for invasive aspergillosis.

Olorofim Aspergillus Infection Study

NCT05101187RecruitingPhase 3
F2G Biotech GmbH
Posted 3/31/2022

Evaluate F901318 (Olorofim) Treatment of Invasive Fungal Infections in Participants Lacking Treatment Options

NCT03583164CompletedPhase 2
F2G Biotech GmbH
Posted 6/6/2018

Ipsen Secures Dual CHMP Approvals for Rare Liver Disease Treatments Following Regulatory Strategy Shift

Drug ApprovalRare DiseaseOrphan Drug
  • Ipsen received CHMP approval for odevixibat under the new brand name Kayfanda for Alagille syndrome, marking the second approval for the same drug after rebranding due to orphan status complications.
  • The company simultaneously secured CHMP recommendation for Iqirvo (elafibranor) as a treatment for primary biliary cholangitis, representing a rare dual approval achievement.
  • Odevixibat demonstrated statistically significant improvements in scratching severity in the ASSERT trial, the world's first phase 3 study completed in Alagille syndrome patients.
  • Both approvals address significant unmet medical needs in rare cholestatic liver diseases, with analysts estimating the PBC market alone could exceed $1.5 billion annually.

Mesoblast's Ryoncil Receives US FDA Orphan Drug Approval, Driving 15% Share Price Surge

Orphan DrugDrug Approval
  • Mesoblast received US FDA orphan drug approval for Ryoncil, granting seven years of market exclusivity and driving a 15% share price increase over one week.
  • The approval significantly strengthens Mesoblast's competitive positioning, with expanded insurance coverage now reaching 104 million US lives.
  • Despite the regulatory milestone, Mesoblast remains unprofitable with A$47.93 million in net losses, though analysts forecast 56.7% annual revenue growth.
  • The company's shares still trade approximately 88% below consensus analyst price targets, suggesting potential for further market adjustments.

FDA Clears BlackfinBio's Gene Therapy Trial for Rare Hereditary Spastic Paraplegia

FDA ApprovalOrphan DrugRare Disease
  • The US FDA has approved BlackfinBio's investigational new drug application for BFB-101, an adeno-associated virus gene therapy targeting hereditary spastic paraplegia type 47 (SPG47).
  • The Phase I/II trial will enroll up to five children with AP4B1-associated SPG47 at Boston Children's Hospital, with recruitment expected to begin by year-end.
  • BFB-101 will be administered via intra-cisterna magna injection to deliver the functional AP4B1 gene copy directly to the central nervous system.
  • The therapy has received FDA orphan drug and rare pediatric disease designations for SPG47, a progressive condition causing lower-limb spasticity and developmental delays.

Catalyst Pharmaceuticals Secures Patent Settlement with Teva, Extending Firdapse Market Exclusivity Until 2035

Orphan DrugRare Disease
  • Catalyst Pharmaceuticals and its licensor SERB S.A. reached a settlement agreement with Teva Pharmaceuticals that prevents Teva from marketing a generic version of Firdapse until February 25, 2035.
  • The settlement resolves patent litigation over Firdapse 10 mg tablets, which treats Lambert-Eaton myasthenic syndrome (LEMS), a rare neuromuscular disorder.
  • Patent disputes with remaining defendants Hetero and Lupin continue, while the agreement must undergo review by the Federal Trade Commission and Department of Justice.
  • The settlement strengthens Catalyst's market position for its flagship orphan drug, following previous regulatory challenges that established important precedents for orphan drug exclusivity.

Ionis Reports Positive Phase 3 Results for Olezarsen in Familial Chylomicronemia Syndrome

Rare DiseaseOrphan Drug
  • Ionis Pharmaceuticals' olezarsen met primary and key secondary endpoints in Phase 3 BALANCE trial for familial chylomicronemia syndrome (FCS), demonstrating significant triglyceride reduction.
  • The antisense therapy showed a favorable safety profile with no major adverse events reported, potentially offering a new treatment option for this rare genetic disorder.
  • Ionis plans to submit regulatory applications in the coming months, positioning olezarsen to potentially become the first approved therapy specifically targeting FCS.

iOnctura Secures €80 Million Series B to Advance PI3Kδ Cancer Therapy Through Phase II Trials

Orphan Drug
  • iOnctura raised €80 million in Series B financing led by Syncona to advance its pipeline of oral cancer treatments targeting neglected and hard-to-treat cancers.
  • Lead asset roginolisib, the first allosteric modulator of PI3Kδ, demonstrated promising efficacy in uveal melanoma with 70% overall survival rate and minimal toxicity in Phase I trials.
  • The funding will accelerate Phase II trials for roginolisib in uveal melanoma and expand development into non-small cell lung cancer and primary myelofibrosis starting late 2024.
  • Second asset cambritaxestat represents the only autotaxin inhibitor in clinical development for highly fibrotic tumors like metastatic pancreatic cancer.

A Study to Assess a PI3Kδ Inhibitor (IOA-244) in Patients with Metastatic Cancers

NCT04328844Active, Not RecruitingPhase 1
iOnctura
Posted 2/25/2020

Dose Escalation Phase 1 Study of IOA-289 in combination with gemcitabine/nab-paclitaxel

2024-515674-27-00Not Yet RecruitingPhase 1
iOnctura SA

FDA Extends Review Timeline for REGENXBIO's Hunter Syndrome Gene Therapy RGX-121

Rare DiseaseOrphan Drug
  • The FDA has extended the review timeline for REGENXBIO's RGX-121 gene therapy for Hunter syndrome, pushing the PDUFA goal date from November 9, 2025 to February 8, 2026.
  • The extension follows REGENXBIO's submission of positive 12-month clinical data for all 13 patients in the pivotal study in response to an FDA information request.
  • RGX-121 would be the first and only potential one-time commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome if approved.
  • The FDA completed pre-license and bioresearch monitoring inspections in August 2025 with no observations and has raised no safety-related concerns during the BLA review.

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