Orphan Drug (original) (raw)

Latest News for: orphan drug

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GSK's momelotinib granted Orphan Drug Designations in the US and EU for VEXAS syndrome (Form 6-K) (GSK plc)

Public Technologies 12 Jun 2026

GSK's momelotinib granted Orphan Drug Designations in the US and EU for VEXAS syndrome.

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Tris Pharma Announces FDA Grant of Orphan Drug Designation for TRN-257 (Oxybate for Extended-Release Oral ...

Nasdaq Globe Newswire 10 Jun 2026

Food and Drug Administration (FDA) has granted Orphan Drug Designation to TRN-257 (oxybate for extended-release oral suspension) for the treatment of ...

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US FDA Grants Orphan Drug and Rare Pediatric Disease Designations to GEn1E Lifesciences’ Dual Signal ...

The Joplin Globe 09 Jun 2026

PALO ALTO, Calif.--(BUSINESS WIRE)--Jun 9, 2026-- ... .

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RedHill's Opaganib Receives FDA Rare Pediatric Disease Designation for Neuroblastoma in Addition to Current Orphan Drug Designation

PR Newswire 09 Jun 2026

This new designation is in addition to opaganib's current neuroblastoma orphan drug ... Opaganib has received Orphan Drug designation from the FDA for the treatment of neuroblastoma and cholangiocarcinoma.

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RedHill’s Opaganib Receives FDA Rare Pediatric Disease Designation for Neuroblastoma in Addition to Current Orphan Drug Designation (Form 6-K) (Redhill Biopharma Ltd)

Public Technologies 09 Jun 2026

Orphan Drug Designation ... This new designation is in addition to opaganib's current neuroblastoma orphan drug ... Opaganib has received Orphan Drug designation from the FDA for the treatment of neuroblastoma and cholangiocarcinoma.

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Attralus Therapeutic Zamubafusp Alfa (AT-02) Receives U.S. FDA Orphan Drug Designation for the Treatment of ...

Nasdaq Globe Newswire 04 Jun 2026

FDA grants orphan drug status to Attralus AT-02 for AL amyloidosis, a novel therapy targeting toxic amyloid removal ... .

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Quoin Pharmaceuticals Announces Japan's MHLW Grants Orphan Drug Designation for QRX003 in

Caledonian Record 04 Jun 2026

-Designation provides regulatory and development incentives in Japan, including up to 10 years of market exclusivity upon approval- ....

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Quoin Pharmaceuticals Announces Japan's MHLW Grants Orphan Drug Designation for QRX003 in Netherton Syndrome

Nasdaq Globe Newswire 04 Jun 2026

-Designation provides regulatory and development incentives in Japan, including up to 10 years of market exclusivity upon approval- ... .

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Quoin Pharmaceuticals Announces Japan's MHLW Grants Orphan Drug Designation for QRX003 in Netherton Syndrome (Form 8-K) (Quoin Pharmaceuticals Ltd)

Public Technologies 04 Jun 2026

Quoin Pharmaceuticals Announces Japan's MHLW Grants Orphan Drug Designation for QRX003 in Netherton Syndrome ... -Follows Orphan Drug Designation, Pediatric Rare Disease Designation, and Fast Track Designation previously granted by the U.S.

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SOTIO Receives U.S. FDA Orphan Drug Designation for SOT106, a Potential Best-in-Class ADC for Sarcoma

Nasdaq Globe Newswire 03 Jun 2026

SOTIO announces the U.S. FDA has granted Orphan Drug Designation (ODD) for the treatment of osteosarcoma to SOT106, the Company's investigational ADC ... .

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SOTIO Receives U.S. FDA Orphan Drug Designation for SOT106, a Potential Best-in-Class ADC for

Caledonian Record 03 Jun 2026

First-in-human study of SOT106 to be initiated in second half of 2026, advancing a differentiated LRRC15 ADC toward early clinical signal ....

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Santhera Announces Orphan Drug and Priority Review Designations for AGAMREE® (Vamorolone) in South Korea

Nasdaq Globe Newswire 02 Jun 2026

SANN) today notes that AGAMREE has been granted Orphan Drug ...

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Pasithea Therapeutics Announces Orphan Drug Designation by FDA of PAS-004 for Treatment of Amyotrophic Lateral ...

Nasdaq Globe Newswire 02 Jun 2026

Pasithea Therapeutics Announces Orphan Drug Designation by FDA of PAS-004 for Treatment of Amyotrophic Lateral Sclerosis (ALS) ... .

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TearSolutions Receives FDA Orphan Drug and Fast Track Designations for Lacripep® in Neurotrophic Keratitis and ...

Business Wire 02 Jun 2026

(“TearSolutions”), a clinical-stage biotechnology company developing novel ophthalmic therapeutics, ...

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Enterome receives U.S. FDA Orphan Drug Designation for EO2463 OncoMimics™ to treat “watch-and-wait” indolent non-Hodgkin lymphoma

Killeen Daily Herald 28 May 2026

Orphan Drug Designation confers regulatory, financial and strategic advantagesEO2463 has shown potentially disease ....