Pyrukynd (mitapivat) is an oral pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
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.
Teplizumab is an intravenously administered anti-CD3 monoclonal antibody in development for the delay of clinical type 1 diabetes (T1D) in at-risk individuals.
Vutrisiran is an injectable RNA interference (RNAi) therapeutic agent under investigation for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
CureSight is a prescription digital therapeutic that combines eye-tracking sensors, image processing algorithms, and real-time remote monitoring under investigation for treatment of amblyopia in children aged 4 to 9 years.
RBX2660 is a microbiota-based investigational product for fecal microbiota transplantation proposed to treat recurrent Clostridoides difficile infection. It is derived from fresh stool from multiple donors; both donors and stool samples are methodically screened. Pooled stool samples are commercially prepared under good manufacturing processes. …
The Luminopia One is a software-only digital therapeutic used with commercially available head-mounted displays intended for home use for improvement in visual acuity in amblyopia patients aged 4-7 years associated with anisometropia and/or with mild strabismus, having received treatment instructions (frequency and duration) as prescribed by a trained eye care professional.
Darvadstrocel is an injectable allogeneic, expanded, adipose-derived mesenchymal stem cell product. It is proposed as an adjunct to standard medical care for treatment-refractory complex perianal fistula in adults with Crohn disease. Darvadstrocel is in phase III clinical trial development for a future regulatory filing with the FDA. It is currently approved under the brand n…
The Melphalan Hepatic Delivery System (M-HDS; proposed U.S. name, Hepzato Kit) is a catheter-based drug delivery and chemofiltration kit for intra-arterial administration of melphalan chemotherapy directly to the liver followed by extracorporeal filtration of hepatic venous blood to reduce melphalan concentration before returning blood to systemic circulation. It is used to p…
The eCoin Peripheral Neurostimulator is a leadless implantable tibial nerve neurostimulator indicated to treat urgency urinary incontinence (UUI) in patients intolerant to or having inadequate response to other more conservative treatments or who have undergone a successful trial of percutaneous tibial nerve stimulation (TNS).
Fexapotide triflutate is an investigational injectable apoptotic agent proposed for the treatment of benign prostatic hypertrophy (BPH).