Nubeqa is a nonsteroidal second-generation androgen receptor inhibitor that competes with androgen by binding to the androgen receptor. It reduces the ability of androgen to promote the growth of prostate cancer cells. Nubeqa is indicated for the treatment of patients with nonmetastatic castration-resistant prostate cancer.
Emerging Technology Reports
The Emerging Technology Report provides regular updates about emerging and potentially disruptive technologies that are likely to have a significant impact in healthcare. This “early warning” report monitors new health technologies from 6 months to 2 years within a regulatory submission and tracks those as they go through clinical trial development and the regulatory approval process.
For each emerging technology, our analysts describe the technology and intended use, its prospects relative to the regulatory process and FDA information, competing technologies, relevant clinical guidelines, high-level look at the evidence such as published abstracts in the medical literature and available meeting abstracts, and available clinical trials.
Brineura (Cerliponase Alfa)
Brineura is recombinant human tripeptidyl peptidase 1 (TPP1) replacement enzyme to slow loss of walking ability in symptomatic pediatric patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Brineura is infused into the cerebrospinal fluid (CSF) via an intraventricular access device implanted in the patient’s head. It is administere…
Brineura (Cerliponase Alfa)
Brineura is recombinant human tripeptidyl peptidase 1 (TPP1) replacement enzyme to slow loss of walking ability in symptomatic pediatric patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Brineura is infused into the cerebrospinal fluid (CSF) via an intraventricular access device implanted in the patient’s head. It is administere…
Onpattro (Patisiran)
Onpattro (patisiran) is an intravenous RNA interference (RNAi) therapeutic agent for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. RNAi is a natural cellular process of gene silencing. Onpattro is designed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to …
Onpattro (Patisiran)
Onpattro (patisiran) is an intravenous RNA interference (RNAi) therapeutic agent for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. RNAi is a natural cellular process of gene silencing. Onpattro is designed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to …
Ultomiris (Ravulizumab-cwvz) for Paroxysmal Nocturnal Hemoglobinuria
Ultomiris is a long-acting humanized murine monoclonal antibody that is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ultomiris binds with high affinity to the complement protein C5, thereby inhibiting its cleavage of C5a and C5b. This inhibition prevents the generation of the terminal complement complex C5b9, preventing complement-mediated hemolys…
Ultomiris (Ravulizumab-cwvz) for Paroxysmal Nocturnal Hemoglobinuria
Ultomiris is a long-acting humanized murine monoclonal antibody that is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Ultomiris binds with high affinity to the complement protein C5, thereby inhibiting its cleavage of C5a and C5b. This inhibition prevents the generation of the terminal complement complex C5b9, preventing complement-mediated hemolys…
Luxturna (Voretigene Neparvovec-rzyl) for Inherited Retinal Dystrophies
Luxturna (voretigene neparvovec-rzyl) is an adenovirus-associated viral vector serotype 2 gene therapy containing the human RPE65 gene for treatment of patients with vision loss due to RPE65-mediated inherited retinal disease. Gene therapy utilizes a viral vector to carry the desired genetic information to target c…
Luxturna (Voretigene Neparvovec-rzyl) for Inherited Retinal Dystrophies
Luxturna (voretigene neparvovec-rzyl) is an adenovirus-associated viral vector serotype 2 gene therapy containing the human RPE65 gene for treatment of patients with vision loss due to RPE65-mediated inherited retinal disease. Gene therapy utilizes a viral vector to carry the desired genetic information to target c…
Libtayo (Cemiplimab)
Libtayo (cemiplimab) is an intravenous programmed death receptor-1 (PD-1) blocking antibody indicated for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation.