Here is information on the latest US FDA approvals, the week of May 15- May 19, 2023. Please subscribe, rate and review. Thank you!
iLet Bionic Pancreas
The FDA has approved components of a “Bionic Pancreas” for patients with type 1 diabetes. The FDA announced the clearance of the Beta Bionics’ iLet ACE Pump and the iLet Dosing Decision Software for people aged 6 years or older with type 1 diabetes. When combined with a compatible FDA-cleared integrated continuous glucose monitor, the trio will form the so called “iLet Bionic Pancreas.”
The Bionic Pancreas uses an adaptive closed-loop algorithm that is initialized only with a user’s body weight and requires no additional insulin dosing parameters. This adaptive algorithm removes the need to manually adjust insulin pump therapy settings and users easily estimate the amount of carbs in their meal, with the algorithm learning over time to respond to users’ individual insulin needs.
VYJUVEK for dystrophic epidermolysis bullosa
The FDA has approved the first-ever topical gene therapy, VYJUVEK (formerly known as B-VEC), for use in dystrophic epidermolysis bullosa (DEB). VYJUVEK will be used to treat wounds in patients 6 months of age and older who have either recessive or dominant DEB a rare skin disease.
DEB is a genetic disorder that affects the skin and, in some cases, other organs. It belongs to a group of conditions that are characterized by extreme skin fragility and blistering. DEB specifically refers to a subtype caused by mutations in the COL7A1 gene.
VYJUVEK uses a nonreplicating HSV-1 vector to deliver the COL7A1 gene directly to skin cells, restoring the COL7 protein fibrils that stabilize skin structure. VYJUVEK is developed by Krystal Biotech and is delivered in a topical gel, applied once a week for 6 months.
EPKINLY for R/R DLBCL
EPKINLY (epcoritamab-bysp) has received approval as the first and only bispecific T cell engager (BiTE) for the treatment of adult patients with relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS), including DLBCL arising from indolent lymphoma, and high-grade B‑cell lymphoma. It is indicated for use after two or more lines of systemic therapy.
In the phase 1/2 EPCORE trial evaluating this agent in 148 patients, an overall response (including a complete or partial response) was observed in 61 percent, and 38 percent achieved complete remission. The median duration of response was 15.6 months. Serious adverse events, as is similar for other bispecific T cell engagers, include cytokine release syndrome (CRS), immune effector cell-associated neurotoxicity syndrome (ICANS), infections, and cytopenias.
Other BiTEs include LUNSUMIO approved in 2022 and indicated for treatment of adult patients with relapsed or refractory follicular lymphoma, TECVAYLI for the treatment of patients with relapsed /refractory multiple myeloma, BLINCYTO, the first approved BiTE, approved in 2014, for acute lymphoblastic leukemia.
MIEBO™ for dry eye disease
Also, last week he FDA has approved perfluorohexyloctane ophthalmic solution (MIEBO™) for the treatment of the signs and symptoms of DED, or dry eye disease.
According to a press release from the manufacturer Bausch + Lomb and Novaliq, this ophthalmic solution is the first-and-only prescription drop approved for DED that directly targets tear evaporation.
B·R·A·H·M·S sFlt-1/ PlGF KRYPTOR Test System
The FDA granted marketing authorization of the B·R·A·H·M·S sFlt-1/ PlGF KRYPTOR Test System (BRAHMS GmbH, Part of Thermo Fisher Scientific) to aid in the risk assessment of certain pregnant women hospitalized for hypertensive disorders. These include preeclampsia, chronic hypertension with or without superimposed preeclampsia, and gestational hypertension, The device is designed to evaluate the progression to preeclampsia with severe features. The test system is an automated immunofluorescent assay that uses Time-Resolved Amplified Cryptate Emission (TRACE) technology for the quantitative detection of placental biomarkers, Placental Growth Factor (PlGF) and Soluble Fms-like tyrosine kinase-1 (sFlt-1) in human serum and plasma.
According to the CDC, preeclampsia is one of the leading causes of maternal morbidity and is estimated to occur in up to 7% of all pregnancies. The hallmarks of preeclampsia are high blood pressure and signs of liver or kidney damage and usually develops after the 20th week of pregnancy.
RINVOQ (upadacitinib) for adults with moderately to severely active Crohn’s disease
Also last week, the FDA approved RINVOQ (upadacitinib) for adults with moderately to severely active Crohn’s disease who have had an inadequate response or intolerance to one or more tumor necrosis factor blockers. RINVOQ is the first approved oral product available to treat moderately-to-severely active Crohn’s disease.
RINVOQ is a Janus kinase (JAK) inhibitor that received its first US approval in 2019.
In addition to the recent approval for Crohn’s disease, RINVOQ is also indicated for use in rheumatoid arthritis, psoriatic arthritis, atopic dermatitis, and ulcerative colitis. Please see prescribing information for the specific details on those indications.
(RSV) prefusion F vaccine candidate, RSVpreF.
The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) voted to support the efficacy and safety of Pfizer’s unadjuvanted bivalent respiratory syncytial virus (RSV) prefusion F vaccine candidate, RSVpreF.
After reviewing the trial data, VRBPAC voted 14 to 0 in favor of the efficacy of RSVpreF and 10 to 0 in favor of the safety. Through active immunization in pregnant persons, RSVpreF prevents medically attended lower respiratory tract disease (MA-LRTD) caused by RSV in infants from birth to 6 months of age.
The vaccine candidate has been granted a PDUFA goal date of August 21, 2023, upon which the FDA will likely decide whether to approve Pfizer’s RSVpreF.
Connexus Direct Data BCI
Finally, the Connexus Direct Data Interface, a brain implant that could help patients with severe paralysis regain their ability to communicate by deciphering their neural signals has received the Breakthrough Device designation from the FDA.
This Designation helps speed up the development and availability of new and innovative medical devices that can treat or diagnose very serious health conditions. When a device is given this designation, it means that it has shown early evidence of being better than the devices currently available. The FDA then works closely with the company to help them get it to patients faster.
A brain-computer interface or BCI is a system that deciphers brain signals and translates them into commands for external technologies. Some examples of BCIs include neuroprosthetics, brain-controlled exoskeletons to regain the ability to walk or move limbs, and assistive communication devices, like the Connexus Direct Data Interface.
Please check back every Monday morning for last week’s approvals so that you can stay up to date.
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