FDA approves Baqsimi nasal powder 3 mg, the first and only nasally administered glucagon to treat severe hypoglycemia in adults and children with diabetes

The FDA has approved Baqsimi (glucagon) nasal powder 3 mg for the treatment of severe hypoglycemia in people with diabetes ages four years and above, Eli Lilly and Company announced today. Baqsimi is the first and only nasally administered glucagon, and it was designed with severe hypoglycemia rescue in mind. It is compact, portable and ready to use (no reconstitution required) in a single, fixed, 3 mg dose. Baqsimi is contraindicated in patients with pheochromocytoma, insulinoma, and known hypersensitivity to glucagon or to any of the excipients in Baqsimi. Allergic reactions have been reported with glucagon and include anaphylactic shock with breathing difficulties and hypotension.

Severe hypoglycemia (very low blood sugar) is a serious medical condition that constitutes an emergency for people with type 1 and type 2 diabetes. It is characterized by altered mental and/or physical functioning that requires assistance from another person for recovery. If untreated, severe hypoglycemia can lead to serious consequences, such as loss of consciousness, seizure, coma and death.

Acquired by Lilly from Locemia Solutions in 2015,Baqsimi is a new formulation of rescue glucagon that builds upon Lilly's diabetes heritage. Dr. Claude Piche, CEO and co-founder of Locemia Solutions, credits co-founder Robert Oringer as the original inspiration behind Baqsimi.

Clinical Overview-In adult patients, Baqsimi had comparable efficacy to injectable glucagon. One hundred percent of pediatric patients (age 4 and above) given Baqsimi met treatment success and 100% of pediatric patients (age 4 and above) given glucagon for injection met treatment success. Common cold with nasal congestion did not impair absorption of Baqsimi. Most common ( greater than 10%) adverse reactions associated with Baqsimi are nausea, vomiting, headache, upper respiratory tract irritation (i.e., rhinorrhea, nasal discomfort, nasal congestion, cough, and epistaxis), watery eyes, redness of eyes, itchy nose, throat and eyes.

Clinical Studies in Adults- Two randomized, multicenter, open-label, 2-period crossover trials compared a 3 mg dose of nasally administered Baqsimi to a 1 mg dose of glucagon for injection for treatment of insulin-induced hypoglycemia. Study 1 included 70 adult patients with type 1 diabetes. Study 2 included 83 adult patients with type 1 or type 2 diabetes; 80 patients were included in the efficacy analysis of Study 2. Primary efficacy measure was the proportion of patients achieving treatment success. Baqsimi demonstrated noninferiority to glucagon for injection in both studies in raising blood glucose after insulin-induced hypoglycemia, with 100% of patients treated with Baqsimi and 100% of glucagon for injection-treated patients achieving treatment success in Study 1, and 98.8% and 100%, respectively, in Study 2. Treatment success was defined as an increase in plasma glucose levels to ? 70 mg/dL or a ?20 mg/dL rise in plasma glucose from nadir within 30 minutes of receiving the study glucagon. In Study 1, the mean nadir blood glucose was 54.5 mg/dL for Baqsimi and 55.8 mg/dL for glucagon for injection. In Study 2, the mean nadir blood glucose was 44.1 mg/dL for Baqsimi and 47.1 mg/dL for glucagon for injection. Spontaneous adverse reactions in adult patients (pooled results for Study 1 and Study 2) for Baqsimi 3 mg (n=153) compared to glucagon for injection 1 mg (n=151) were nausea (26.1% vs. 33.8%), headache (18.3% vs. 9.3%), vomiting (15.0% vs. 13.9%) and upper respiratory tract irritation (rhinorrhea, nasal discomfort, nasal congestion, cough, and epistaxis) (12.4% vs. 1.3%). A patient-solicited questionnaire identified (treatment-emergent) nasal and ocular symptoms for Baqsimi 3 mg compared to glucagon for injection 1 mg, including watery eyes (58.8% vs. 2.0%), nasal congestion (42.5% vs. 6.0%), runny nose (34.6% vs. 0%), nasal itching (39.2% vs. 4.6%), itchy eyes (21.6% vs. 1.3%), redness of eyes (24.8% vs. 2.6%), sneezing (19.6% vs. 0.7%), itching of throat (12.4% vs. 1.3%) and itching of ears (3.3% vs. 0.7%). Subjects were asked to report whether they had the symptom after glucagon administration. The frequency was based on severity increase over baseline.

Clinical Studies in Pediatric Patients- A randomized, multicenter study evaluated the pharmacokinetics/pharmacodynamics (PK/PD), safety, and efficacy of Baqsimi compared with glucagon for injection in children and adolescents aged 4 to less than 17 years with type 1 diabetes (N=48) divided into 3 cohorts. Young children (ages 4 to less than 8 years, n=18) and children (ages 8 to less than 12 years, n=18) were randomized 2:1 to receive either Baqsimi 2 mg or 3 mg at visit 1 and the alternative Baqsimi dose at visit 2; or weight-based glucagon for injection at a single study visit. Adolescents (ages 12 to less than17, n=12) were randomized 1:1 to receive Baqsimi 3 mg or glucagon for injection 1 mg with crossover to alternate glucagon at dosing visit 2. Insulin was used to reduce blood glucose levels, and glucagon was administered after glucose reached <80 mg/dL. Following insulin-induced glucose reduction, 100% of patients achieved treatment success with nasally administered Baqsimi compared to 100% of patients treated with glucagon for injection. Treatment success was defined as the proportion of participants achieving an increase in plasma glucose of greater than 20 mg/dL from the nadir glucose concentration within 30 minutes of glucagon dosing. The mean nadir blood glucose across different age cohorts was 67-73 mg/dL for BaqsimiI and 69-72 mg/dL for glucagon for injection. Spontaneous adverse reactions in pediatric patients for Baqsimi 3 mg (n=36) compared to weight-based glucagon for injection (n=24) were vomiting (30.6% vs. 37.5%), headache (25.0% vs. 12.5%), nausea (16.7% vs. 33.3%) and upper respiratory tract irritation (nasal discomfort, nasal congestion, and sneezing) (16.7% vs. 0%).

Clinical Study in Nasal Congestion-A randomized, single center, open-label, repeated-measures, parallel-design, phase 1 study examined the safety and PK/PD of Baqsimi in otherwise healthy adult participants with nasal congestion resulting from common cold. Adult patients in cohort 1 (n=18) received 2 doses of Baqsimi 3 mg: one while experiencing nasal congestion and another after recovery from cold symptoms. Adult patients in cohort 2 (n=18), who also had colds with nasal congestion, received a single dose of Baqsimi 3 mg 2 hours after treatment with the decongestant oxymetazoline. Blood glucagon and glucose concentrations were measured before and at 5-, 10-, and 30-minute intervals until 180 minutes after Baqsimi administration. Common cold with nasal congestion tested with or without use of decongestant did not impair absorption of Baqsimi.

People can sign up for direct updates on Baqsimi availability, patient and caregiver resources, and affordability options at BAQSIMI.com. Baqsimi is expected to be stocked in retail pharmacies within one month.