In the dynamic landscape of pharmaceuticals and healthcare, staying updated on the latest developments in Orphan Drug Designation is of paramount importance. As we delve into the month of September 2023, the world of orphan drugs continues to evolve, bringing with it both exciting breakthroughs and critical alerts. These advancements have the potential to impact the lives of individuals affected by rare diseases profoundly. In this article, we have covered most of the recent news and alerts surrounding Orphan Drug Designation by leading regulatory agencies FDA, EU, and PMDA, among others, shedding light on the innovations, challenges, and regulatory updates that are shaping the future of orphan drug development and accessibility.
| Post Date | Title | Drug/Therapy | Indication/Disease | Key Updates and Announcements | Press Release |
| September 7, 2023 | Intensity Therapeutics Receives Orphan Drug Designation for the three key ingredients in INT230-6 for the Treatment of Soft Tissue Sarcoma | INT230-6 | Soft Tissue Sarcoma | INT230-6, Intensity’s lead proprietary investigational product candidate, is designed for direct intratumoral injection. INT230-6 was discovered using Intensity’s proprietary DfuseRx℠ technology platform. The drug is composed of two proven, potent anti-cancer agents, cisplatin and vinblastine, and a penetration enhancer molecule (SHAO) that helps disperse potent cytotoxic drugs throughout tumors for diffusion into cancer cells. These agents remain in the tumor resulting in a favorable safety profile. In addition to local disease control, direct killing of the tumor by INT230-6 releases a bolus of neoantigens specific to the patient’s malignancy, leading to engagement of the immune system and systemic anti-tumor effects. | Orphan Drug Designation for the three key ingredients in INT230-6 for the Treatment of Soft Tissue Sarcoma |
| September 7, 2023 | Harmony Biosciences Announces US Food & Drug Administration Orphan Drug Designation For Pitolisant For Treatment of Idiopathic Hypersomnia | Pitolisant | Idiopathic Hypersomnia | Harmony is currently evaluating the efficacy and safety of pitolisant in adult patients with IH in the Phase 3 registrational INTUNE study, a double-blind, placebo-controlled, randomized withdrawal study. Topline study results are anticipated in the fourth quarter of 2023 following enrollment completion nine months ahead of plan. | Orphan Drug Designation For Pitolisant For Treatment of Idiopathic Hypersomnia |
| September 7, 2023 | MaaT Pharma Announces European Medicines Agency Granted MaaT033 Orphan Drug Designation Aiming to Improve Overall Survival in Patients Undergoing Hematopoietic Stem Cell transplantation | MaaT033 | Hematopoietic Stem Cell transplantation | MaaT033, a donor-derived, high-richness, high-diversity oral Microbiome Ecosystem TherapyTM containing anti-inflammatory ButycoreTM species, is currently being developed as an adjunctive therapy to improve overall survival in patients receiving HSCT and other cellular therapies. It aims to ensure optimal microbiota function and to address a larger patient population in a chronic setting. MaaT033 has been granted Orphan Drug Designation by the European Medicines Agency (EMA). | Orphan Drug Designation Aiming to MaaT033 for the Patients Undergoing Hematopoietic Stem Cell Transplantation |
| September 6, 2023 | FDA grants Orphan Drug Designation to CAN10 for treatment of systemic sclerosis | CAN10 | Treatment of Systemic Sclerosis | The CAN10 antibody strongly binds IL1RAP and simultaneously blocks the function of the signaling molecules IL-1, IL-33 and IL-36, which play key roles in several autoimmune and inflammatory diseases. CAN10 has previously shown promising effects in several models of such diseases, including the lead indications systemic sclerosis and myocarditis. A clinical phase I trial investigating the safety and tolerability of CAN10 in healthy volunteers and psoriasis patients is ongoing. Up to 80 subjects may be included in the trial, and initial data from the trial are expected in 2024. | Orphan Drug Designation to CAN10 for treatment of systemic sclerosis |
| September 5, 2023 | Krystal Biotech Announces Orphan Drug Designation Granted to KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency | KB408 | Alpha-1 Antitrypsin Deficiency | KB408 is an inhaled (nebulized) formulation of Krystal Biotech’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment of AATD. | Orphan Drug Designation to KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency |