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Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog

The Clinical Trials Grants Program provides funding for clinical trials that evaluate the safety and efficacy of potential treatments for rare diseases to help move promising treatments through clinical development. Relative to other areas of medicine (e.g.,

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KFA-115 by Novartis for Melanoma: Likelihood of Approval

Pharmaceutical Technology

KFA-115 is under clinical development by Novartis and currently in Phase I for Melanoma. It provides drugs for the treatment of cancer, cardiovascular diseases, dermatological conditions, neurological disorders, ophthalmic and respiratory diseases, hematologic diseases, solid tumors, immune disorders, and infections, among others.

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KFA-115 by Novartis for Malignant Mesothelioma: Likelihood of Approval

Pharmaceutical Technology

KFA-115 is under clinical development by Novartis and currently in Phase I for Malignant Mesothelioma. The company offers generic medicines and biosimilars through Sandoz. GlobalData’s report assesses how KFA-115’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks.

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Iptacopan hydrochloride by Novartis for Paroxysmal Nocturnal Hemoglobinuria: Likelihood of Approval

Pharmaceutical Technology

Iptacopan hydrochloride is under clinical development by Novartis and currently in Phase III for Paroxysmal Nocturnal Hemoglobinuria. The company offers generic medicines and biosimilars through Sandoz. Novartis conducts research in various disease areas through The Novartis Institutes for BioMedical Research (NIBR).

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Iptacopan hydrochloride by Novartis for Glomerulonephritis: Likelihood of Approval

Pharmaceutical Technology

Iptacopan hydrochloride is under clinical development by Novartis and currently in Phase III for Glomerulonephritis. The company offers generic medicines and biosimilars through Sandoz. Novartis conducts research in various disease areas through The Novartis Institutes for BioMedical Research (NIBR).

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(Lopinavir + ritonavir) by Douglas Pharmaceuticals for Genital Warts (Condylomata Acuminata): Likelihood of Approval

Pharmaceutical Technology

(Lopinavir + ritonavir) is under clinical development by Douglas Pharmaceuticals and currently in Phase I for Genital Warts (Condylomata Acuminata). Its investigational medicinal products comprise R-107, R-131 and R-147. The company offers consumer, prescription and automation products.

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(Lopinavir + ritonavir) by Douglas Pharmaceuticals for Cervical Intraepithelial Neoplasia (CIN): Likelihood of Approval

Pharmaceutical Technology

(Lopinavir + ritonavir) is under clinical development by Douglas Pharmaceuticals and currently in Phase I for Cervical Intraepithelial Neoplasia (CIN). Its investigational medicinal products comprise R-107, R-131 and R-147. The company offers consumer, prescription and automation products.