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.

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

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.

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

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.

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:

FDA Grants Orphan Drug Designation to Zenocutuzumab‑zbco for NRG1+ CCA The FDA has granted Orphan Drug Designation to zenocutuzumab-zbco (Bizengri) for advanced NRG1 fusion-positive cholangiocarcinoma (CCA).

The @fda.gov has granted #OrphanDrug Designation to zenocutuzumab-zbco (#Bizengri®) for adults with advanced unresectable or metastatic neuregulin 1 (#NRG1) gene fusion-positive #Cholangiocarcinoma (#CCA), according to a recent press release.

Read here: https://bit.ly/4apGNlR

#RareDisease #MedSky

Bayer／BlueRock、iPSC由来細胞治療OpCT-001が網膜色素変性でFDAオーファンドラッグ指定取得 | STELLANEWS.LIFE STELLANEWS.LIFE（ステラニュース・ライフ）は、科学や技術、医薬品分野における最新の研究成果や発見 | STELLANEWS.LIFE（ステラニュース・ライフ）は、科学や技術、医薬品分野における最新の研究成果や発見

FDA grants Orphan Drug Designation to BlueRock Therapeutics’ investigational cell therapy OpCT-001 for the treatment of retinitis pigmentosa

stellanews.life/technology/8...

#RetinitisPigmentosa #iPSC #CellTherapy #Bayer #BlueRock #FDA #OrphanDrug #ClinicalTrials

Every quarter, orphan drug designations change. Every quarter, some hospitals miss savings because they didn't update their tracking. Don't be one of them.

#340B #OrphanDrug

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).

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).

Genesipre’s gene therapy GENE202 has received Orphan Drug Designation for methylmalonic acidemia (MMA), a rare pediatric disease.
#News #GeneTherapy #RareDisease #OrphanDrug #MMA #Biotech

New year, same mission: helping CAHs, RRCs, and SCHs maximize orphan drug savings. What 340B questions are on your mind for 2026?

#340B
#OrphanDrug

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).

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

Announcement of a new publication on non-oncologic orphan drug approvals, focusing on evidence types and approval timelines worldwide.

📢 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

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.

#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

MHRA set to overhaul UK’s rare disease drug regulatory pathway The medicines regulator will aim to take a more flexible licensing approach for the research and manufacture of rare disease drugs in the UK.

MHRA set to overhaul the UK’s rare disease drug regulatory pathway with the aim of establishing a more flexible licensing approach

www.europeanpharmaceuticalreview.com/news/267591/...

#pharma #mhra #raredisease #orphandrug

FDA Orphan Designation Supports Ofirnoflast as Novel MDS Therapy US FDA Orphan Drug Designation supports continued development of ofirnoflast (HT-6184) for myelodysplasic syndromes (MDS).

The @fda.gov granted #OrphanDrug Designation to #Ofirnoflast (HT-6184) for the treatment of #MyelodysplasticSyndromes (#MDS), recognizing the agent’s potential to address a key upstream mechanism of inflammation-driven bone marrow failure.

Read more: https://bit.ly/48PwfvV

#RareDisease #HT6184

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

Orphan drugs (which target patient populations in the US under 200,000) are still key drivers for pharma top-lines #orphandrug

It's interesting to see that for some companies, the share of revenue from orphan drugs is declining. LOEs are also driving absolute declines for $BMY and $PFE

GeoVax CEO David Dodd shares his management philosophy in the face of substantial change in the healthcare environment.
$GOVX
#biotech #biotechnology #science #GeoVax #Gedeptin #CancerTherapy #InnovativeMedicine #Biotech #TargetedTherapy #Immunotherapy #OrphanDrug #ClinicalTrials #CancerTreatment

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.

#biotech #Servier #FragileXsyndrome #FXS #rarediseases #KaerusBioscience #FRAXAResearchFoundation #FXStherapies #orphandrug #rarepaediatricdrugdesignation #FDA #neurodevelopmentaldisorder #raremovementdisorder #lifesciencesventurecapital #Medicxi #BKchannelmodulatorprogramme
zurl.co/EPSas

If approved this October, patients may soon have a new needle‑free option to manage swelling
episodes quickly and confidently.

#HereditaryAngioedema #HAE #RareDisease #Sebetralstat #Ekterly #OrphanDrug #KalVista #OnDemandTherapy #BioNews #AngioedemaNews

Bluejay begins phase 3 trial of brelovitug for hepatitis D - PharmaTimes First patient enrolled in AZURE-2 study comparing brelovitug to Hepcludex

#Bluejay #BluejayTherapeutics #phase3trial #brelovitug #BJT778 #hepatitisD #AZURE2study #CHD #hepatitisDvirus #cirrhosis #livercancer #viralhepatitis #liverdisease #BreakthroughTherapydesignation #FDA #PRIMEdesignation #Orphanstatus #OrphanDrug #EMA
pharmatimes.com/news/bluejay...

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

Vanda Pharmaceuticals stock rises after FDA orphan drug designation Investing.com -- Vanda Pharmaceuticals (NASDAQ:VNDA) stock rose 6% after the company announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its VGT-1849B treatment for polycythemia vera (PV). VGT-1849B is a selective peptide nucleic acid-based JAK2 inhibitor designed to target the treatment of PV, a chronic myeloproliferative disorder that affects an estimated 44 to 57 per 100,000 people in the United States. The condition is characterized by excessive red blood cell production and increased release of pro-inflammatory cytokines. The company’s treatment approach differs from existing JAK inhibitors on the market by specifically targeting JAK2 mRNA with high precision, potentially reducing off-target effects that can lead to increased toxicity. More than 95% of PV patients harbor the JAK2 V617F gain-of-function mutation that leads to aberrant JAK2 production. Currently available JAK inhibitors such as Jakafi, Inrebic, Ojjaara, and Vonjo are not solely selective to JAK2, which can result in binding to off-target kinases. Vanda’s approach aims to reduce JAK2 protein production without affecting other JAK family members, potentially offering a more favorable safety profile. VGT-1849B utilizes a novel backbone chemistry called OliPass Peptide Nucleic Acid that improves cell permeability and affinity for RNA by introducing cationic lipid moieties onto nucleobases. The Orphan Drug Designation provides various benefits to drug developers addressing rare medical conditions, including tax credits for clinical trials and exemption from user fees. This article was generated with the support of AI and reviewed by an editor. For more information see our T&C. 3rd party Ad. Not an offer or recommendation by Investing.com. See disclosure here or remove ads. Should you invest $2,000 in VNDA right now? First, check if it's included in one of this month's AI-powered stock strategies for ProPicks AI. Investing.com created these strategies to identify the most exciting trading opportunities currently in the market. The stocks that made the cut could produce monster returns in the coming years, like ViaSat and Sapiens , both up over 60%+ each in Q2 of 2025 alone. Is VNDA one of them?

Click Subscribe #VandaPharmaceuticals #FDA #OrphanDrug #StockMarket #Investing

EATRIS is a partner of the World Orphan Drug Congress - one of the largest gatherings in #RareDisease & #OrphanDrug development.

Final call: the 35% early bird offer ends tomorrow, 29 Aug!

More here👉 eatris.eu/events/world-orphan-drug... #RareDiseases #WODC2025 #DrugDevelopment

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

Soligenix stock soars after FDA grants orphan drug designation Investing.com -- Soligenix (NASDAQ:SNGX) stock surged 88% after the FDA granted orphan drug designation to dusquetide, the active ingredient in SGX945, for the treatment of Behçet’s Disease. The designation follows the FDA’s review of recent Phase 2a clinical results that demonstrated biological efficacy and safety in patients with the rare autoimmune condition. Orphan drug status provides Soligenix with seven years of market exclusivity upon final FDA approval, along with potential financial benefits including government grants for clinical trials, waiver of FDA user fees for a potential New Drug Application, and certain tax credits. Behçet’s Disease affects up to 18,000 people in the U.S., 50,000 in Europe, 350,000 in Turkey, and as many as 1 million people worldwide. The condition is characterized by oral aphthous ulcers and is considered difficult to treat and incurable. "The FDA’s decision to grant orphan drug designation to the SGX945 program signifies an important step for Soligenix as we continue to advance the program and adds significantly to the existing intellectual property estate surrounding this novel technology," said Christopher J. Schaber, President and CEO of Soligenix. Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases with unmet medical needs. The orphan drug designation is designed to encourage companies to develop therapies for conditions affecting fewer than 200,000 people in the United States. This article was generated with the support of AI and reviewed by an editor. For more information see our T&C. With SNGX making headlines, savvy investors are asking: Is it truly valued fairly? In a market full of overpriced darlings, identifying true value can be challenging. InvestingPro's advanced AI algorithms have analyzed SNGX alongside thousands of other stocks to uncover hidden gems. These undervalued stocks, potentially including SNGX, could offer substantial returns as the market corrects. In 2024 alone, our AI identified several undervalued stocks that later surged by 30 or more. Is SNGX poised for similar growth? Don't miss the opportunity to find out.

Click Subscribe #Soligenix #FDA #OrphanDrug #StockMarket #Investing

We're a partner of the World Orphan Drug Congress Europe - one of the largest events in #RareDisease & #OrphanDrug development.
27–29 Oct | Amsterdam | 250+ speakers | 2,000+ attendees

More info & register (with "EAT20" for 20% off) here👉 eatris.eu/events/world-orphan-drug...

Mustang Bio stock soars after FDA grants orphan drug designation Investing.com -- Mustang Bio Inc (NASDAQ:MBIO) stock surged over 78% after the U.S. Food and Drug Administration granted orphan drug designation to its MB-101 treatment for recurrent diffuse and anaplastic astrocytoma and glioblastoma, rare forms of brain cancer. The designation provides Mustang Bio with several benefits, including tax credits for clinical trials and seven years of market exclusivity if the treatment receives regulatory approval. The FDA’s designation covers a broader indication than what was originally proposed by the company. MB-101 consists of IL13Ra2-targeted CAR T-cells designed specifically to target these aggressive brain cancers. In an ongoing Phase 1 trial published in Nature Medicine, the treatment showed promising results with 50% of patients achieving stable disease or better outcomes. Notably, two patients achieved complete responses lasting 7.5 and 66+ months respectively. The study evaluated 57 patients total, with the two complete responses occurring in a cohort of three patients who had the highest levels of intratumoral CD3+ T cells before treatment. Mustang Bio is also developing MB-109, a combination therapy using MB-101 with MB-108, an oncolytic virus treatment for malignant glioma that previously received orphan drug designation. This combination approach uses MB-108 to modify the tumor microenvironment to potentially enhance MB-101’s effectiveness. The company noted that further development of the MB-109 program depends on securing additional funding or establishing a strategic partnership. Mustang licenses MB-101 from City of Hope and MB-108 from Nationwide Children’s Hospital. Phase 1 clinical trials for both treatments continue enrolling patients at City of Hope and The University of Alabama at Birmingham. This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

Click Subscribe #MustangBio #StockMarket #FDAGrants #OrphanDrug #Investing

Hematology Weekly News – June 23rd 2025 🔬🩸 Hematology Updates: DARZALEX, CAR NK Breakthroughs, and Triplet AML Therapy Momentum From CHMP approvals and pediatric trial expansions to […] The post Hematology Weekly News – June 23rd 2025 appeared first on LucidQuest Ventures.

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Hematology Weekly News – June 23rd 2025 🔬🩸 Hematology Updates: DARZALEX, CAR NK Breakthroughs, and Triplet AML Therapy Momentum From CHMP approvals and pediatric trial expansions to […] The post Hematology Weekly News – June 23rd 2025 appeared first on LucidQuest Ventures.

ICYMI: LucidQuest Views >>> Hematology Weekly News – June 23rd 2025 #News #orphandrug #pharmainnovation #clinicaltrials Comment below!

Hematology Weekly News – June 23rd 2025 🔬🩸 Hematology Updates: DARZALEX, CAR NK Breakthroughs, and Triplet AML Therapy Momentum From CHMP approvals and pediatric trial expansions to […] The post Hematology Weekly News – June 23rd 2025 appeared first on LucidQuest Ventures.

LucidQuest Views >>> Hematology Weekly News – June 23rd 2025 #News #orphandrug #pharmainnovation #clinicaltrials Comment below!