FDA Approves Key Component for Common Childhood Cancer Treatment

July 06, 2021 - FDA recently approved Jazz Pharmaceutical’s Rylaze as a component of a chemotherapy regimen to treat the most common childhood cancer

Specifically, Rylaze was approved to treat lymphoblastic leukemia and lymphoblastic lymphoma in adult and pediatric patients allergic to E. coli-derived asparaginase products commonly used for cancer treatment.

The only other FDA-approved drug for patients with these allergic reactions has been in a global shortage for years, the agency highlighted. 

“It is extremely disconcerting to patients, families and providers when there is a lack of access to critical drugs for treatment of a life-threatening, but often curable cancer, due to supply issues,” Gregory Reaman, MD, associate director for pediatric oncology in the FDA’s Oncology Center of Excellence, said in the announcement.

“Today’s approval may provide a consistently sourced alternative to a pivotal component of potentially curative therapy for children and adults with this type of leukemia,” Reaman continued.

FDA based its approval on a study of 102 patients who either had a hypersensitivity to E. coli-derived asparaginases or experienced silent inactivation. The primary outcome of the study was whether patients achieved and maintained a certain level of asparaginase activity.

Researchers found that Rylaze elicited the target level of asparaginase activity in 94 percent of patients. 

The most common side effects of the drug included hypersensitivity reactions, pancreatic toxicity, blood clots, hemorrhage, and liver toxicity. 

Previously, Rylaze received fast track and orphan drug designations. FDA conducted this review under Project Orbis, an initiative of the FDA Oncology Center of Excellence. 

FDA Approves Direct Biologics Phase 1/2 Trial Application for COVID-19 Treatment

Direct Biologics recently announced that FDA has approved the application for a Phase 1/2 trial for the use of ExoFlo in the treatment for mild-to-moderate COVID-19. 

This is the company’s third FDA-approved investigational new drug (IND) trial of ExoFlo and the first IND FDA approved to investigate the use of extracellular vesicles to treat the virus. 

"The FDA's approval to initiate our third IND trial of ExoFlo for treatment of COVID-19 represents a significant milestone for Direct Biologics. Our momentum initiating ongoing clinical trials signifies our commitment to delivering solutions for patients who have been affected in multiple ways by COVID-19,” Mark Adams, co-founder and chief executive officer of Direct Biologics, said in the announcement.

Direct Biologics will commence with a Phase 1/2 trial for mild-to-moderate COVID-19, called EMOTE COVID-19, at the end of 2021. The trial will be double-blinded, placebo-controlled, and randomized. 

The company previously completed two clinical trials evaluating ExoFlo to treat COVID-19-related acute respiratory distress syndrome in inpatient and ICU settings.

Additionally, Direct Biologics recently received approval to proceed with a Phase 1/2 trial for post-acute COVID-19 syndrome and chronic post-COVID-19 syndrome.

FDA Approves Angel Medical Systems Cardiac Detection Monitor

Angel Medical Systems recently announced that FDA approved the second-generation AngelMed Guardian device, the first implantable cardiac detection monitor and patient-warning system for acute coronary syndrome (ACS) events, including silent heart attacks. 

The AngelMed Guardian is implanted by a cardiologist during a low-risk, outpatient surgical procedure and includes ease-of-use adaptations and an updated long-life battery that could double its life. 

The device records the heart’s electrical activity 24 hours a day, looking for electrical changes that can indicate a possible ACS event. 

"The improved AngelMed Guardian device will have a meaningful effect on the current standard of patient cardiology care for ACS events. Our dedicated team and supporting physicians have worked tirelessly to bring this disruptive technology to market," Brad Snow, chief executive officer at Angel Medical Systems, said in the announcement. 

Every 40 seconds, someone in the US suffers a myocardial infarction or heart attack.

Michael Gibson, MD at Boston Clinical Research Institute, stated that patients who have had a prior ACS event are often at high risk of a recurrent event.

The AngelMed Guardian System demonstrated the ability to identify any early signs of an ACS event more effectively than patients’ symptoms alone and in patients who do not experience symptoms at all.