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FDA Approvals Roundup: Kloxxado, Farxiga, Ferriprox

Posted 05 May 2021 | By Renee Matthews 

FDA Approvals Roundup: Kloxxado, Farxiga, Ferriprox

A weekly update on new drug approvals and indications from the US Food and Drug Administration (FDA).
New approvals
Higher-dose Kloxxado nasal spray cleared for countering opioid overdose
Hikma’s Kloxxado (naloxone hydrochloride nasal spray) has been approved for treating opioid overdose. Its indicated dose of 8 mg is notably higher than the previously approved doses of 2 and 4 mg for other naloxone nasal spray products.
Kloxxado was approved through the 505(b)(2) pathway, whereby an applicant can rely on safety and effectiveness findings from either the FDA or in published literature for a previously approved product that is similar to the application drug. In the case of Kloxxado, the applicant used the agency’s safety and effectiveness findings for Narcan (injectable naloxone hydrochloride) to support its application. It also provided supporting pharmacokinetic data demonstrating the opioid antagonist’s safety and efficacy.
New indications
Farxiga use expanded for reducing risks in patients with chronic kidney disease
AstraZeneca’s Farxiga (dapagliflozin tablet) has received an expanded indication for lowering the risk of kidney function decline, kidney failure, cardiovascular (CV) death, and hospitalization for heart failure in adults with chronic kidney disease and risk of progression. 
Approval of the sodium-glucose cotransporter 2 (SGLT2) inhibitor was based on efficacy findings in the interventional, multicenter, randomized Dapa-CKD study. In all, 4,304 patients were randomized 1:1 to receive Farxiga or placebo and were followed for a median of 28.5 months. The primary composite endpoint included reduction in kidney function, progression to kidney failure, or CV- or kidney-related death. A total of 197 patients in the study arm had at least one of the composite endpoint events, compared with 312 patients in the placebo arm. Among study arm patients, 100 were hospitalized for heart failure or had a CV-related death, compared with 138 patients in the placebo arm.
Farxiga received fast track, breakthrough therapy and priority review designations for the current indication.
The SGLT2 inhibitor was originally approved in 2014 to improve glycemic control in adults with type 2 diabetes in addition to diet and exercise.
Ferriprox extended for adults, kids with iron overload due to SCD and other anemias
Chiesi’s Ferriprox (deferiprone) has received a new indication for treating transfusional iron overload due to sickle cell disease (SCD) or other anemias, including thalassemia, in adults and children aged 3 years or older.
Ferriprox was originally approved in 2011 as a therapy for patients with transfusional iron overload due to thalassemia syndromes for whom chelation therapy was not adequate.
Current approval of the iron-chelating agent was based on efficacy findings in the randomized, controlled, interventional FIRST study in patients from the indicated population. They were randomized to receive Ferriprox or Novartis’ Desferal (deferoxamine mesylate) and monitored for change in liver iron concentration for 12 months after baseline. The findings demonstrated Ferripox’s noninferiority to Desferal. Data from an extension study in patients receiving Ferriprox confirmed that liver iron levels decreased progressively for up to 3 years (baseline, 14.93 mg/g dry weight [dw]; 1 year, 12.30 mg/g dw; 2 years, 11.19 mg/g dw; and 3 years, 10.45 mg/g dw).
Pembrolizumab picks up extension for HER2-positive gastric cancer
Merck’s Keytruda (pembrolizumab), in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, has been granted a new indication for the first-line treatment of patients with locally advanced, unresectable, or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma.
The approval was based on results in a prespecified interim analysis of the first 264 patients of the ongoing KEYNOTE-811 trial. Patients in the multicenter, double‑blind, placebo‑controlled trial had not received previous systemic therapy for metastatic disease. They were randomized 1:1 to receive pembrolizumab or placebo with a combination therapy. Overall response rate was 74% in the pembrolizumab arm and 52% in the placebo arm. Median duration of response was 10.6 versus 9.5 months, respectively.
The review used the real-time oncology review pilot and the assessment aid. The application was granted priority review.
Keytruda, a programmed death receptor-1 blocking antibody, was originally approved in 2014 as a therapy for patients with unresectable or metastatic melanoma, and is used to treat a number of cancers, including classical Hodgkin lymphoma and cervical and non-small cell lung cancers.


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