Release Date: 22 September 2021
US FDA has granted orphan drug designation to Ganaxolone for treatment in TSC. Ganaxolone is a helpful allosteric modulator of GABAA receptors, is an investigational product being established in intravenous and oral formulations intended...
Release Date: 21 September 2021
US FDA has granted the orphan drug designation to sotigalimab (APX005M) for the treatment of soft tissue sarcoma. Sotigalimab is planned to stimulate antitumor immune response and to target CD40, which is a key co-stimulatory receptor th...
Release Date: 21 September 2021
US FDA has granted orphan drug designation for NFX-179 for the treatment of cutaneous neurofibromatosis type 1. NFX-179 is a mitogen-activated protein kinase kinase (MEK) inhibitor. NFX-179 is a “soft” (metabolically labile) ...
Release Date: 20 September 2021
US FDA has given orphan drug designation for intravitreal injection (ADX-2191) for the treatment of retinitis pigmentosa (RP). As of now, no approved drugs are available for the treatment of this rare illness. RP is characterized by reti...
Release Date: 20 September 2021
Autobahn Therapeutics, a biotechnology company announces that US FDA has granted orphan drug designation for ABX-002, for the treatment of X-linked adrenoleukodystrophy (X-ALD).
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Release Date: 19 September 2021
US FDA has designated Orphan Drug designation to AER-901 (inhaled imatinib) for the treatment of pulmonary arterial hypertension. Imatinib is currently available as an oral formulation to treat several types of cancers. AER-901 is an inv...
Release Date: 19 September 2021
US FDA has granted Orphan Drug Designation to glycosylated cannabinoid prodrug VBX-100 for the treatment of patients with pediatric ulcerative colitis. Vitality Biopharma Inc, is looking forward to accelerate their research to improve sa...
Release Date: 18 September 2021
US FDA has announced Orphan Drug Designation to aviptadil (RLF-100) for the treatment of sarcoidosis. Aviptadil is an inhaled, synthetic formulation of vasoactive intestinal peptide (VIP). Aviptadil is shown to preserve surfactant produc...
Release Date: 18 September 2021
US FDA has designated Orphan Drug Designation VO659, an investigational antisense oligonucleotide gene-silencing therapy for Huntington’s disease. VO659 such as, Antisense oligonucleotides are artificially created nucleotides that ...
Release Date: 17 September 2021
Aldeyra Therapeutics receives Orphan Drug Designation from US FDA for ADX-2191 to treat retinitis pigmentosa. ADX-2191 is an intravitreal formulation of methotrexate that inhibits dihydrofolate reductase, an enzyme involved in cellular r...
Release Date: 17 September 2021
Medpacto’s Vactosertib Receives FDA Orphan Drug Designation to the combination therapy of Vactosertib and PD-L1 inhibitors for treating osteosarcoma. Osteosarcoma is a tumor that occurs in bone or osteoid tissue, such as cartilage ...
Release Date: 16 September 2021
US FDA has granted an orphan drug designation (ODD) to alrizomadlin (APG-115), an MDM2-p53 inhibitor, for the treatment of stage 2b to 4 melanoma. Alrizomadlin in combination with a PD-1 blockade advances antitumor activity by activating...
Release Date: 16 September 2021
US FDA has granted both rare pediatric disease designations and orphan drug designation to LX1004 for CLN2 disease which is also known as late infantile Batten disease. Lexeo Therapeutics has completed a Phase 1 clinical trial of LX1004 ...
Release Date: 15 September 2021
Aldeyra Therapeutics earns Orphan Drug Designation from US FDA for ADX-2191 (methotrexate for intravitreal injection) for the treatment of primary vitreoretinal lymphoma (PVRL). ADX-2191 inhibits dihydrofolate reductase, an enzyme involv...
Release Date: 15 September 2021
European Medicines Agency (EMA) has granted Orphan Drug Designation to INZ-701 for the treatment of ABCC6 Deficiency, an investigational enzyme replacement therapy (ERT), was granted Orphan Drug Designation by the US FDA and the EMA for ...