Pharmafile Logo

News and Press Releases

SOM Biotech secures clear registrational path for SOM3355 in Huntington’s disease after FDA End‑of‑Phase 2 Meeting

20 October 2025 -- Barcelona, Spain -- SOM Biotech, a clinical-stage company dedicated to the discovery and development of innovative therapies for rare central nervous system disorders, announces today that it...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: October 20, 2025

SOM Biotech
Baldiri Reixac, 4 (08028) Barcelona, Spain

Pharming Group announces U.S. FDA acceptance and Priority Review of supplemental New Drug Application forleniolisib in children with APDS aged 4 to 11 years

If approved, leniolisib will be first and only treatment indicated for children with activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome (APDS), a rare primary immunodeficiency Decision based on positive data from...

Category: Biotechnology, Drug Discovery, Other, Pharmaceutical
Posted: October 1, 2025

Pharming Group NV
info@pharming.com
Pharming Group N.V. Vondellaan 47 2332 AA Leiden The Netherlands

Sanofi’s SAR402663 earns fast track designation in the US for neovascular age-related macular degeneration

Designation earned for a one-time intravitreal gene therapy with the potential to eliminate treatment burden for people living with neovascular age-related macular degeneration Neovascular or “wet” age-related macular degeneration can...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: September 11, 2025

Sanofi
One Onslow Street, Guildford, Surrey, GU1 4YS, UK

Iksuda Therapeutics receives FDA IND clearance for IKS014

FDA decision will enable Iksuda to expand its ongoing clinical trial in the US, Australia and Singapore Preliminary data from an ongoing dose-escalation study of IKS014 has shown meaningful clinical activity across...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: July 1, 2025

Iksuda
The Biosphere, Draymans Way, Newcastle Helix Newcastle upon Tyne NE4 5BX

SolasCure receives FDA Fast Track Designation for Aurase Wound Gel to treat calciphylaxis ulcers

Recognises the potential of Aurase Wound Gel to improve outcomes for individuals with calciphylaxis ulcers, a rare and life-threatening condition Represents a new indication for Aurase Wound Gel, currently in...

Category: Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: June 16, 2025

SolasCure
wellington house East Rd, Petersfield, Cambridge CB1 1BH

FDA Grants OKYO Pharma Fast Track Designation to Urcosimod for Neuropathic Corneal Pain

1 May 2025 -- London, UK and New York, US -- OKYO Pharma Limited, an ophthalmology-focused bio-pharmaceutical company which is developing urcosimod (formerly called OK-101) to treat NCP, an ocular...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: May 1, 2025

OKYO Pharma
info@okyopharma.com
OKYO Pharma Floor 4, 14/15 Conduit St London W1S 2XJ

Fondazione Telethon Submits US Biological License Application for Etuvetidigene Autotemcel Gene Therapy for the Treatment of Wiskott-Aldrich Sydrome

11 March 2025 -- Milan, Italy -- Fondazione Telethon, an Italian biomedical charity focused on rare genetic diseases, announced that it has submitted the Biologics License Application (BLA) for the...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: March 11, 2025

Fondazione Telethon
info@telethon.it
Via Poerio 14 - 20129 Milano

OKYO Pharma Files for Fast Track Designation with FDA for Urcosimod to Treat Neuropathic Corneal Pain

10 March 2025 -- London, UK and New York, US -- OKYO Pharma Limited, a clinical-stage biopharmaceutical company developing innovative therapies for the treatment of neuropathic corneal pain, a severe...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: March 10, 2025

OKYO Pharma
info@okyopharma.com
OKYO Pharma Floor 4, 14/15 Conduit St London W1S 2XJ

Bioxodes obtains Orphan Drug Designation for hemorrhagic stroke therapeutic candidate in US and Europe

FDA, EMA grant BIOX-101 ODD status to treat intracerebral hemorrhagic stroke (ICH) ODD status may expedite drug candidate development and approvals BIOX-101 interim Phase 2a data in ICH imminent. 5...

Category: Drug Discovery, Other, Pharmaceutical
Posted: March 5, 2025

Bioxodes
info@bioxodes.com
Parc d'activités Aéropole Rue Santos-Dumont, 1 6041 Gosselies, Belgique

Tiziana Life Sciences Files Investigational New Drug Application with FDA for ALS Phase 2 Clinical Trial

4 March 2025 -- New York, US -- Tiziana Life Sciences, Ltd, a biotechnology company developing breakthrough immunomodulation therapies with its lead development candidate, intranasal foralumab, a fully human, anti-CD3...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: March 4, 2025

Tiziana Life Sciences
info@tizianalifesciences.com
Tiziana Life Sciences 14/15 Conduit Street London W1S 2XJ United Kingdom

Boehringer’s nerandomilast meets primary endpoint in Phase III study FIBRONEER-ILD, in progressive pulmonary fibrosis

Topline data from FIBRONEER™-ILD show that the investigational compound nerandomilast met its primary endpoint, which was the absolute change from baseline in forced vital capacity [mL] at week 52 versus...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: February 10, 2025

Boehringer Ingelheim
Binger Strasse 173 55216 Ingelheim am Rhein

Opella reaches study milestone for Cialis

21 January 2025 -- Paris, France -- Opella, Sanofi’s Consumer Healthcare business, announced that the US Food and Drug Administration (FDA) has lifted a clinical hold on its planned actual...

Category: Clinical Trials, Other, Pharmaceutical
Posted: January 21, 2025

Sanofi
One Onslow Street, Guildford, Surrey, GU1 4YS, UK

PureTech Receives FDA Fast Track Designation for LYT-200 in Acute Myeloid Leukemia (AML)

Single agent and combination data from Phase 1b AML/MDS trial presented at ASH 2024 showed potential of LYT-200 to serve broad range of patients across various lines of treatment LYT-200...

Category: Clinical Trials, Drug Discovery, Other, Pharmaceutical
Posted: January 9, 2025

PureTech Health
info@puretechhealth.com
PureTech Health 6 Tide Street Boston, MA 02210

PTC Therapeutics Announces FDA Acceptance for Filing of NDA for Sepiapterin for the Treatment of Pediatric and Adult Phenylketonuria Patients

1 October 2024 -- New Jersey, US -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for the treatment of...

Category: Biotechnology, Drug Discovery, Pharmaceutical
Posted: October 1, 2024

PTC Therapeutics
info@ptcbio.com
PTC Therapeutics Inc. 100 Corporate Court South Plainfield, NJ 07080-2449

PureTech-Invented KarXT Receives U.S. Food and Drug Administration Approval for the Treatment of Schizophrenia in Adults

Milestone triggers payments to PureTech totaling $29 million under agreements with Royalty Pharma and PureTech’s Founded Entity, Karuna Therapeutics, which was acquired by Bristol Myers Squibb in March 2024, and...

Category: Other, Pharmaceutical
Posted: September 27, 2024

PureTech Health
info@puretechhealth.com
PureTech Health 6 Tide Street Boston, MA 02210