Here is information on the latest US FDA approvals, the week of June 26 – June 30, 2023. Please subscribe, rate and review this podcast. Thank you!
Lantidra® for Type 1 Diabetes
The FDA has approved Lantidra, manufactured by CellTrans Inc., for the treatment of type 1 diabetes. Lantidra (donislecel-jujn) is the first allogeneic pancreatic islet cellular therapy derived from deceased donor pancreatic cells. The treatment is specifically approved for patients with type 1 diabetes who cannot achieve target glycated hemoglobin levels due to repeated episodes of severe hypoglycemia despite intensive diabetes management and education. Type 1 diabetes requires multiple daily injections of insulin or continuous infusion using a pump. Some individuals with type 1 diabetes struggle to manage their daily insulin requirements to prevent hyper or hypoglycemia. Lantidra is administered through an infusion into the hepatic portal vein. Additional infusions may be necessary depending on the patient’s response to the initial dose. The primary mechanism of action of Lantidra is believed to be the secretion of insulin by the infused allogeneic islet beta cells. In certain patients with type 1 diabetes, these cells can produce sufficient insulin, eliminating the need for insulin injections or pump usage to control blood sugar levels. In a study involving 30 participants who received at least one infusion and a maximum of three infusions, 5 participants did not achieve insulin independence. However, the remaining 25 patients achieved at least one year of insulin independence, with 10 participants being able to go more than five years without insulin. Lantidra has been associated with severe adverse events and requires the use of ongoing immunosuppressive medications to maintain islet cell viability.
https://www.fda.gov/media/169920/download
Ngenla® for Pediatric Growth Hormone Deficiency
The FDA has approved Ngenla (somatrogon-ghla) for the treatment of patients 3 years of age or older with growth failure due to inadequate secretion of endogenous growth hormone. Ngenla is administered through weekly injections, which is different from most currently available treatments that are given daily. Other long-acting agents for this condition include Novo Nordisk’s Sogroya, which was approved for adults in 2020, and Ascendis’ Skytrofa, the first long-acting agent approved for use in children in 2020. Ngenla is now the second long-acting agent approved for use in children. The approval of Ngenla was based on results from a Phase 3 study involving 224 treatment-naive pediatric patients with growth hormone deficiency. The study demonstrated non-inferiority compared to somatropin, using annual height velocity at 12 months as a measure. The FDA approval follows a complete response letter issued by Pfizer in January 2022, without publicly stated reasons. Growth hormone deficiency is considered a rare disease, affecting approximately one in 4,000 to 10,000 children.
https://labeling.pfizer.com/ShowLabeling.aspx?id=19642&format=pdf
Roctavian® for Hemophilia A
The FDA has approved Roctavian, a gene therapy based on an adeno-associated virus vector, for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5. The generic name for Roctavian is valoctocogene roxaparvovec-rvox suspension for intravenous infusion. Hemophilia A is a rare genetic bleeding disorder caused by a mutation in the gene responsible for producing factor VIII, a protein essential for blood clotting. This disorder primarily affects males. The effectiveness of Roctavian was established based on results from a cohort of 112 patients who were followed up for at least 3 years after treatment. Following the infusion, the mean annualized bleeding rate decreased from 5.4 bleeds per year at baseline to 2.6 bleeds per year. The standard treatment for severe hemophilia A usually involves the use of Factor VIII replacement therapy or an antibody-based medication to improve blood clotting ability and reduce the likelihood of bleeding. Roctavian, on the other hand, is a one-time gene therapy administered as a single dose through intravenous infusion. The FDA approval was granted to Biomarin.
https://www.fda.gov/media/169937/download
Rystiggo® for Generalized Myasthenia Gravis
FDA approval has been granted to Rystiggo (rozanolixizumab-noli) under the Priority Review designation for the treatment of generalized myasthenia gravis (gMG) in adult patients who are positive for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibodies. The approval is based on the pivotal Phase 3 MycarinG study in gMG, a large phase 3 study that demonstrated significant improvements in various outcomes, such as breathing, talking, swallowing, and mobility. Rystiggo is administered as a subcutaneous infusion of a humanized IgG4 monoclonal antibody that binds to the neonatal Fc receptor (FcRN), leading to a reduction in circulating IgG. It is the only FDA-approved treatment in adults for both anti-AChR and anti-MuSK antibody-positive gMG, which are the two most common subtypes of gMG. According to the manufacturer, UCB, this medication will be available commercially in the US during the 3rd quarter of 2023.
https://www.ucb-usa.com/RYSTIGGO-prescribing-information.pdf
Skyclarys® for Friedrich’s Ataxia
Skyclarys (omaveloxolone) has become the first FDA-approved treatment for Friedreich’s ataxia. According to the manufacturer, Reata Pharmaceuticals, the treatment is now available to patients following a brief delay due to a production process-related impurity. The treatment was initially approved in February 2023 for use in patients aged 16 years and older. Friedreich’s ataxia is an inherited neurodegenerative disorder that affects approximately 5,000 patients in the US. It is typically diagnosed during adolescence and causes progressive loss of muscle strength and coordination, leading to wheelchair confinement and a shortened lifespan.
https://hcp.skyclarys.com/docs/skyclarys_us_prescribing_information/
Atorvaliq® Atorvastatin Liquid Suspension
The FDA has approved Atorvaliq (atorvastatin calcium), the first and only FDA-approved liquid suspension of atorvastatin for the treatment of high cholesterol and various risk factors for heart disease and stroke. The medication, manufactured by CMP Pharma, Inc., is a liquid oral suspension approved for patients 10 years of age and older. According to the manufacturer, Atorvaliq is bioequivalent to the tablet dose of atorvastatin and has a stable shelf life of 2 years.
https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/213260s000lbl.pdf
Please check back every Monday morning for last week’s approvals so that you can stay up to date.
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