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Disc`s Rare Disease Drug Hit By FDA Rejection
14 Feb 2026 //
PRESS RELEASE
Biohaven CEO Calls Recent FDA Decisions `A Systemic Problem`
13 Feb 2026 //
FIERCE BIOTECH
HanchorBio Receives FDA Orphan Drug for HCB101 in Gastric Cancer
13 Feb 2026 //
PR NEWSWIRE
PTC Withdraws US Approval Filing For Duchenne Drug Translarna
13 Feb 2026 //
FIERCE PHARMA
Ultragenyx Lays Off 10% Of Workers As FDA Stalls Approval Plan
13 Feb 2026 //
FIERCE BIOTECH
EU Approves Amgen`s Uplizna for Generalized Myasthenia Gravis
13 Feb 2026 //
PR NEWSWIRE
ImmuneSensor Doses First Patient in Phase 1b IMSB-301 AGS Trial
12 Feb 2026 //
GLOBENEWSWIRE
Biomarin Bet On Amicus To Ease Voxzogo`s Decline. Will It Work?
12 Feb 2026 //
BIOSPACE
MPox Orphan Drug Designation Application Filed for NV-387
12 Feb 2026 //
PHARMIWEB
PacBio Joins Ihope Initiative For Long-Read Genomic Sequencing
12 Feb 2026 //
GLOBENEWSWIRE
PTC Therapeutics Provides Regulatory Update On Translarna
12 Feb 2026 //
PR NEWSWIRE
Satellos Doses First Patient in Phase 2 SAT-3247 DMD Study
12 Feb 2026 //
PHARMIWEB
Vyome Files for VT-1953 Orphan Drug Status
11 Feb 2026 //
BUSINESSWIRE
Intrabio Details Positive Levacetylleucine Trial
11 Feb 2026 //
BUSINESSWIRE
FDA Approves Optune Pax for Locally Advanced Pancreatic Cancer
11 Feb 2026 //
BUSINESSWIRE
Precision Biosciences Gets FDA Nod For PBGENE-DMD In DMD
11 Feb 2026 //
BUSINESSWIRE
THX Pharma, Biocodex Sign Deal for Batten, Gaucher, NPC Diseases
10 Feb 2026 //
BUSINESSWIRE
Aardvark Submits FDA & IRB-Approved Trial Protocol FOR ARD-101
10 Feb 2026 //
GLOBENEWSWIRE
REGENXBIO Announces Regulatory Update On RGX-121 BLA For MPS II
10 Feb 2026 //
PR NEWSWIRE BIO
Inmune Bio Progresses CORDStrom For UK Marketing Approval in RDEB
10 Feb 2026 //
GLOBENEWSWIRE
Measles Orphan Drug Designation Application Filed for NV-387
10 Feb 2026 //
PHARMIWEB
Polaryx Reveals SOTERIA Pre-Clinical Data At 22nd Worldsymposium
10 Feb 2026 //
GLOBENEWSWIRE
Mannkind Enrolls First Patient In INHALE-1ST Pediatric Study
09 Feb 2026 //
GLOBENEWSWIRE
Sanofi`s Rilzabrutinib Designated Breakthrough Therapy In US
09 Feb 2026 //
GLOBENEWSWIRE
AB Science Receives FDA MUMS Status For Masivet In Canine Mast
09 Feb 2026 //
GLOBENEWSWIRE
Oxford-Harrington Center Advances Friedreich`s Ataxia Therapies
09 Feb 2026 //
GLOBENEWSWIRE
Solid Biosciences Reports Favorable FDA Type C Meeting Outcome
09 Feb 2026 //
GLOBENEWSWIRE
Ultragenyx Gene Therapy Demonstrates Long-Term Efficacy
09 Feb 2026 //
BIOSPACE
Sarepta Challenges Prompt Search For Advancements In DMD Therapy
09 Feb 2026 //
BIOSPACE
FDA Rejects Regenxbio`s Hunter Syndrome Gene Therapy
09 Feb 2026 //
FIERCE BIOTECH
Parkinson`s Disease Surge Predicted In India By 2026 Symposium
08 Feb 2026 //
ECONOMICTIMES
Zydus Gets USFDA Orphan Drug Designation For Desidustat
07 Feb 2026 //
INDPHARMAPOST
Uniqure Halts Mid- To High-Dosing For AAV Gene Therapy
06 Feb 2026 //
FIERCE BIOTECH
Synaptixbio: Biotech Hails Reauthorization Of Rare Disease Drug
06 Feb 2026 //
PHARMIWEB
Chiesi Global Rare Diseases Strengthens Rare Disease Community
06 Feb 2026 //
PHARMIWEB
Lucerastat`s Registration Path Positions It As Potential First
06 Feb 2026 //
GLOBENEWSWIRE
Biogen Bets HD Spinraza Can Outmuscle Roche`s Oral Rival
06 Feb 2026 //
FIERCE PHARMA
RARE Investors Lead Ultragenyx Pharma Securities Fraud Lawsuit
06 Feb 2026 //
PHARMIWEB
FDA Approves Label Update For Kite`s Yescarta® In Cns Lymphoma
06 Feb 2026 //
BUSINESSWIRE
Aucta Pharma Readies PYQUVI™ Oral Suspension Launch For Rare
06 Feb 2026 //
PR NEWSWIRE BIO
India Confronts Severe Shortage In Registered Blood Stem Cell
06 Feb 2026 //
PHARMABIZ
Zydus Secures USFDA Orphan Drug Status For Desidustat In Sickle
06 Feb 2026 //
ECONOMICTIMES
Zydus` Desidustat Receives USFDA Orphan Drug Designation
06 Feb 2026 //
EXPRESSPHARMA
Denali Unveils LSD Advances at 2026 WORLD Symposium
05 Feb 2026 //
GLOBENEWSWIRE
Zenocutuzumab?Zbco Granted FDA Orphan Drug Designation
05 Feb 2026 //
PR NEWSWIRE
Worldsymposium Showcases Tralensidase Alfa For Sanfilippo
05 Feb 2026 //
BUSINESSWIRE
Amgen Thumbs-Down on FDA’s ask, Tavneos Sales Continue
05 Feb 2026 //
FIERCE PHARMA
Atossa Pharma Sustains (Z)-Endoxifen Dominance In DMD
05 Feb 2026 //
PR NEWSWIRE
Chiesi Global Rare Diseases Strengthens Rare Disease Community
05 Feb 2026 //
GLOBENEWSWIRE
Cognition Therapeutics Extends Zervimesine Access Program
05 Feb 2026 //
GLOBENEWSWIRE
Longeveron Supports Mikaela Naylon Act & Rare Pediatric Disease
05 Feb 2026 //
GLOBENEWSWIRE
Mirum Pharma Announces Health Canada Authorization of Livmarli
05 Feb 2026 //
BUSINESSWIRE
Sarepta Saga Has `Gone On Too Long` As Competitors Catch Up
05 Feb 2026 //
BIOSPACE
Cumberland Pharma Granted FDA Fast Track For Ifetroban
04 Feb 2026 //
PR NEWSWIRE
JCR Pharma Unveils Investigational Lysosomal Disorder Treatments
04 Feb 2026 //
BUSINESSWIRE
Mezzion Pharma Applauds Give Kids A Chance Act Enactment
04 Feb 2026 //
PR NEWSWIRE
Sarepta Secures CTA for SRP-1005 Trial in Huntington`s Disease
04 Feb 2026 //
PHARMIWEB
Zevra Therapeutics Unveils Positive Real-World Miplyffa Data
04 Feb 2026 //
GLOBENEWSWIRE
FDA`s Rare Pediatric Voucher Program Revived By Funding Bill
04 Feb 2026 //
FIERCE PHARMA
Government Proposes Duty Drop On Rare Disease Drugs
03 Feb 2026 //
ECONOMICTIMES
