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vanjohn10.bsky.social
CEO David Dodd shares GeoVax' strategy for generating value driven by clinical development milestones.
$GOVX
#biotech #biotechnology #science #GeoVax #Gedeptin #CancerTherapy #Oncology #InnovativeMedicine #Biotech #TargetedTherapy #Immunotherapy #OrphanDrug #ClinicalTrials #CancerTreatment
September 6, 2025 at 6:27 PM
thebabaklab.bsky.social
The @fda.gov has approved the first gene therapy for children with Sanfilippo syndrome type A.

Link in the comments 👇

#ClinicalMondays #GeneTherapy #SanfilippoSyndrome #Pediatrics #OrphanDrug #FastTrack #BreakthroughTherapy
September 21, 2026 at 3:54 AM
eurordis.bsky.social
📢 New #IRDiRC study on global #orphandrug approvals: it sheds light on the persistent global delays patients face in accessing innovative therapies for rare diseases.

🔗 https://go.eurordis.org/IRDiRCNewPublication
Announcement of a new publication on non-oncologic orphan drug approvals, focusing on evidence types and approval timelines worldwide.
December 3, 2025 at 8:00 AM
umc-pv.bsky.social
Rare diseases and #OrphanDrug monitoring represent a complex and evolving landscape in healthcare and pharmaceutical research.

This #RareDiseaseDay, learn how a new era of innovation must be complemented by post-marketing surveillance in #UppsalaReports:
Orphan Drugs: Hope for rare disease communities
Orphan drug monitoring emerges as a critical lifeline for rare diseases, offering hope to patients with uncommon medical conditions.
uppsalareports.org
February 28, 2026 at 10:10 AM
aboutscience.bsky.social
The XXII National Conference on Pharmaceuticals focused on medicine assessment, new regulations, collaboration, #orphandrug evaluation, and AIFA’s reorganization to optimize pricing and reimbursement. | journals.aboutscience.eu/index.php/gr...
February 26, 2025 at 10:39 AM
checkrare.bsky.social
Johanna Rossell, Senior Vice President and General Manager of Rare Diseases at Sumitomo Pharma America, discusses best practices for navigating challenges of the orphan drug market.

checkrare.com/navigating-t...

#CheckRare #RareDisease #OrphanDrug
May 23, 2025 at 3:36 PM
theevidencebase.bsky.social
Curing the incurable: Can the ORPHAN Cures Act mitigate the #IRA’s impact on #orphandrug development? | Becaris Publishing becarispublishing.com/digital-cont...
Curing the incurable: Can the ORPHAN Cures Act mitigate the IRA’s impact on orphan drug development? | Becaris Publishing
becarispublishing.com
February 27, 2025 at 10:04 AM
4040rec.bsky.social
#Bluejay #BluejayTherapeutics #phase3trial #brelovitug #BJT778 #hepatitisD #AZURE2study #CHD #hepatitisDvirus #cirrhosis #livercancer #viralhepatitis #liverdisease #BreakthroughTherapydesignation #FDA #PRIMEdesignation #Orphanstatus #OrphanDrug #EMA
pharmatimes.com/news/bluejay...
Bluejay begins phase 3 trial of brelovitug for hepatitis D - PharmaTimes
First patient enrolled in AZURE-2 study comparing brelovitug to Hepcludex
pharmatimes.com
September 10, 2025 at 12:03 PM
scge.bsky.social
In this video of the Meet the Expert Series, Deanna Portero and Stefano Benvenuti discuss alternative business models for non-commercially viable diseases.

Check out this thoughtful discussion at: www.youtube.com/watch?v=1WeB...

#RareDisease #OrphanDrug #Biotech
Meet the Expert: Deanna Portero and Stefano Benvenuti
YouTube video by SCGE Outreach
www.youtube.com
October 22, 2025 at 1:44 PM
d-and-d-world.bsky.social
Genesipre’s gene therapy GENE202 has received Orphan Drug Designation for methylmalonic acidemia (MMA), a rare pediatric disease.
#News #GeneTherapy #RareDisease #OrphanDrug #MMA #Biotech
Genesipre’s rare disease therapy gets orphan drug status in the US and Europe
The gene therapy is being developed to treat a rare pediatric metabolic disease, methylmalonic acidemia (MMA).
druganddeviceworld.com
January 21, 2026 at 5:39 PM
rarediseaseadvisor.bsky.social
Kynurenic acid (#FS2) has received Orphan Drug Designation (#ODD) from the @fda.gov for the treatment of idiopathic pulmonary fibrosis (#IPF), as recently announced by BirchBioMed.

Learn more: https://bit.ly/4pr61VU

#RareDisease #OrphanDrug #IdiopathicPulmonaryFibrosis #MedSky
FDA Grants Orphan Drug Designation to Kynurenic Acid (FS2) for IPF
Kynurenic acid (FS2) has received Orphan Drug Designation from the FDA for the potential treatment of idiopathic pulmonary fibrosis (IPF).
bit.ly
December 9, 2025 at 5:21 PM
rarediseaseadvisor.bsky.social
#ADX324, a next-generation small interference (#siRNA) therapeutic for hereditary angioedema (#HAE), has received #OrphanDrug Designation from the US FDA and entered phase 3 development, with the first patient dosed in the #STOPHAE trial.

Read more: https://bit.ly/43hyVPt

#RareDisease #MedSky
First Patient Dosed in Phase 3 STOP-HAE Trial of ADX-324
ADX-324, a next-generation siRNA therapeutic for HAE, has entered phase 3 development with the first patient dosed in the STOP-HAE trial.
bit.ly
November 7, 2025 at 4:50 PM
pharmoinstitute.bsky.social
In case you missed it, our latest whitepaper provides a practical guide to closing the rare disease product timeline gap; evidence transportability, external control arms, sample-size preservation in real-world evidence, and where to invest early. buff.ly/rlXt6uf

#EUHTA #JCA #OrphanDrug #RWE
September 9, 2026 at 10:02 AM
pharmoinstitute.bsky.social
If you're developing a therapy for a rare disease, we recommend you check out our new whitepaper, a practical guide to closing that gap: evidence transportability, external control arms, sample-size preservation in #RWE, and where to invest early. buff.ly/rlXt6uf

#EUHTA #JCA #OrphanDrug
August 21, 2026 at 10:00 AM
theminingnews.bsky.social
🚀 Zacks Small Cap Research - PLX: Elfabrio Estimates Up
🔎 Read the full update:
➡️https://theminingnews.com/zacks-small-cap-research-plx-elfabrio-estimates-up/
#Protalix #Protalix #ElfabrioP#PRX115r#PLXe#FabryDiseaset#GoutTreatmentu#OrphanDrug##PhaseIIr#RELEASETrialu#BRIGHTStudyP#EMAA#CHMP#EUApprov
August 14, 2026 at 2:46 AM
pharmalot.bsky.social
Pharmalittle: We’re reading about a Lilly access program, a Gilead court victory over a 'novel' theory, and more news.. statnews.com/pharmalot/20... #pharma #Lilly #Gilead #obesity #HIV #medicines #CVS #raredisease #orphandrug
Pharmalittle: We're reading about a Lilly access program, a Gilead court victory, and more
Eli Lilly confirmed that it will allow more patients to apply for early access to retatrutide, an unapproved obesity drug
statnews.com
August 4, 2026 at 1:28 PM
lumanity.bsky.social
From 2028, orphan drugs face EU JCA built on RCTs, validated endpoints, and standard of care rare diseases rarely have. Read our new whitepaper exploring how to build a defensible evidence package anyway.

lumanity.com/perspectives...

#EUHTA #JCA #orphandrug
Text: Whitepaper. Preparing for Universal Orphan Drug JCA Submissions. Lumanity logo.
July 28, 2026 at 11:00 AM
Orphan Drug Designation Sends Shares of this Dual-Listed Life Science Therapy Company Soaring
#OrphanDrug designation, or ODD, is valuable on both a humanitarian and a profitability basis. And this company is seeing shares rally following news that it was granted the designation for the treatment of #Pericarditis, the swelling, and irritation of…
dlvr.it
February 15, 2024 at 7:27 PM
fazencapital.bsky.social
Plus Therapeutics Jumps After FDA Grants Orphan-Drug Status: Plus Therapeutics received FDA orphan-drug status on Apr 8, 2026; orphan designation gives 7 years of U.S. exclusivity, changing commercial… 👈 Read full analysis #PlusTherapeutics #OrphanDrug #FDAApproval #HealthcareInvestment #PharmaNews
Plus Therapeutics Jumps After FDA Grants Orphan-Drug Status
Plus Therapeutics received FDA orphan-drug status on Apr 8, 2026; orphan designation gives 7 years of U.S. exclusivity, changing commercial optionality for PSTV.
dlvr.it
April 8, 2026 at 5:35 PM
fazencapital.bsky.social
SCYNEXIS Acquires SCY-770 for Kidney Disease: SCYNEXIS announced on Mar 31, 2026 the acquisition of SCY-770 for a rare kidney disease; regulatory incentives apply for populations under 200,000 in the U.S. 👈 Read full analysis #SCYNEXIS #KidneyDisease #RareDisease #OrphanDrug #Pharmaceuticals
SCYNEXIS Acquires SCY-770 for Kidney Disease
SCYNEXIS announced on Mar 31, 2026 the acquisition of SCY-770 for a rare kidney disease; regulatory incentives apply for populations under 200,000 in the U.S.
dlvr.it
March 31, 2026 at 11:04 AM
fazencapital.bsky.social
Pharming Wins EU Backing for Immunodeficiency Therapy: CHMP issued a positive opinion on Mar 27, 2026 (Seeking Alpha). EC decision typically arrives within 67 days; EU orphan status grants 10 years… 👈 Read full analysis #Pharming #Immunodeficiency #EUBacking #OrphanDrug #HealthcareInnovation
Pharming Wins EU Backing for Immunodeficiency Therapy
CHMP issued a positive opinion on Mar 27, 2026 (Seeking Alpha). EC decision typically arrives within 67 days; EU orphan status grants 10 years exclusivity.
dlvr.it
March 27, 2026 at 4:35 PM
arnatarthera.bsky.social
Arnatar Therapeutics has officially emerged from stealth.

With a $52M Series A led by Eight Roads & 3E Bioventures and ‪@fda.gov‬ #OrphanDrug + #RarePediatricDisease designations for ART4 in #AlagilleSyndrome, we’re advancing a new era of #RNABasedTherapies.‬

Learn more: https://bit.ly/4m39ec1
August 25, 2025 at 7:00 PM
dmdwarrior.com
Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy

#dmd #duchenne #dyne #dyne-251 #exon51 #exon51skipping #exonskipping #ema #orphandrug
Dyne Therapeutics Receives European Medicines Agency (EMA) Orphan Drug Designation for DYNE-251 in Duchenne Muscular Dystrophy Dyne Therapeutics, today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD). Today, Dyne Therapeutics, a clinical-stage company dedicated to developing life-changing treatments for individuals with genetically driven neuromuscular diseases, announced that DYNE-251 has been designated as an orphan drug by the European Commission (EC) for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being tested in DMD patients who are susceptible to exon 51 skipping in the Phase 1/2 DELIVER global clinical study.
April 24, 2025 at 12:19 PM
umc-pv.bsky.social
Rare diseases and #OrphanDrug monitoring represent a complex and evolving landscape in healthcare and pharmaceutical research. Payal Gajbhiye shares how the new era of innovation must be complemented by post-marketing surveillance in #UppsalaReports👇
Orphan Drugs: Hope for rare disease communities
Orphan drug monitoring emerges as a critical lifeline for rare diseases, offering hope to patients with uncommon medical conditions.
uppsalareports.org
April 17, 2025 at 2:52 PM
4040rec.bsky.social
#biotech #Servier #FragileXsyndrome #FXS #rarediseases #KaerusBioscience #FRAXAResearchFoundation #FXStherapies #orphandrug #rarepaediatricdrugdesignation #FDA #neurodevelopmentaldisorder #raremovementdisorder #lifesciencesventurecapital #Medicxi #BKchannelmodulatorprogramme
zurl.co/EPSas
Servier bets $450m on Fragile X syndrome drug
Servier has pushed its emerging neurology pipeline into the clinical trials stage by licensing a drug for Fragile X syndrome from the UK's Kaerus.
pharmaphorum.com
September 17, 2025 at 8:18 AM