Recent FDA Approvals to Target Chronic Disease Management

A treatment for hemophilia is the latest FDA approval seeking to improve chronic disease management.

FDA recently approved Sevenfact for the treatment of hemophilia A or B with inhibitors in adults and adolescents 12 years of age or older.

Hemophilia A or B is a congenital bleeding disorder caused by a deficiency of Coagulation Factor VIII or IX, respectively. Individuals with this condition may bleed for a longer time than others after injury or surgery. 

“Today’s approval provides another treatment option for the control of bleeding episodes in adults and adolescents with hemophilia who have developed inhibitors,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in the announcement. 

Sevenfact is a unique treatment option for patients with the chronic disease. The product is the first hemophilia treatment containing an active ingredient obtained from rabbits genetically engineered to produce a protein for blood coagulation.

“This approval is an example of our efforts to advance safe biotechnology innovations to support patient health,” Marks stated.

Nearly 20,000 individuals are living with hemophilia in the US, according to the CDC. This FDA approval is important because individuals with inhibitors may not respond to factor replacement therapy. Any bleeding episodes are currently managed either on demand of prophylaxis. 

“Based on a comprehensive analysis of the scientific evidence, the FDA determined that the rDNA construct inserted in the rabbit’s genomic DNA is safe for the animal and for the people handling the rabbits, and is effective in causing Human Factor VII (hFVII) protein to be expressed in the rabbits’ milk,” said Steven M. Solomon, DVM, MPH, director of the FDA’s Center for Veterinary Medicine. “The FDA has also determined under the National Environmental Policy Act that approval of the application will have no significant impact on the environment.”

A recent clinical study of 27 patients with hemophilia A or B with inhibitors determined the safety and efficacy of Sevenfact over a 12-hour period after the initial dose was given. This included the treatment of 465 mild or moderate patients and three severe bleeding episodes. 

Eighty-six percent of patients were treated successfully with the lower dose of 75mcg/kg and a higher dose of 225mcg/kg. The three severe bleeding episodes were treated with the higher dose.

Bristol Myers Scores FDA Approval for Celgene’s MS Drug Ozanimod

FDA recently approved Zeposia (ozanimod), a multiple sclerosis (MS) drug developed by Celgene, according to a recent announcement from the Bristol-Myers Squibb Company.

Last year, Bristol-Myers acquired Celgene in a $74 billion merger deal. Ozanimod was one of the many drugs that Celgene had its eyes on.

“With the FDA approval of Zeposia, appropriate patients with relapsing forms of multiple sclerosis will have another oral treatment option with meaningful efficacy to help address the disease’s hallmark relapses and brain lesions,” Samit Hirawat, MD, chief medical officer at Bristol Myers Squibb, said in the announcement

“Zeposia has substantial clinical potential, and we are well positioned with our heritage in transformational science to ensure this innovative compound ultimately benefits as many patients as possible.”

Zeposia will address relapsing forms of MS including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. FDA approved the MS drug after originally rejecting the drug, solidifying the Bristol Myers- Celgene merger. 

FDA Approves Prescription Digital Therapeutic for Chronic Insomnia

Pear Therapeutics recently announced that FDA approved Somryst, the first prescription digital therapeutic (PDT) to treat adult patients with chronic insomnia. 

Patients 22 years of age and older can use Somryst over a nine-week period. It is the first product submitted through FDA’s traditional 510(k) pathway. The treatment was also reviewed as part of FDA’s Software Precertification Pilot Program to help build and test FDA’s Digital Health Precertification Working Model 1.0.

“More than 30 million adults suffer from chronic insomnia, which can seriously impact one’s quality of life and can lead to depression, suicidality, hypertension, and even heart attacks. Treatment options for chronic insomnia are limited as most available sleep medications are only recommended for short term use, can lead to problems with tolerance, and can have unwanted side-effects, including next-day cognitive impairments,” Charles M. Morin, MD, PhD, professor of psychology, director the Sleep Research Centre at Université Laval in Quebec City, said in the announcement.

Somryst is the first PDT to receive authorization from FDA following Pear reSET and reset-o and the only FDA-authorized PTD therapeutic that delivered guidance recommended first-line treatment for chronic insomnia. 

Somryst will provide neurobehavioral interventions, focusing on Cognitive Behavioral Therapy for insomnia (CBT) and sleep restrictions to greatly improve insomnia symptoms. The PDT is personalized to the patients’ needs, such as their ability to set a sleep window.

FDA Approves Roche’s Biomarker Test for At-Risk HPV Infections

In mid-March, Roche announced that FDA approved its CINtec PLUS Cytology as the first biomarker-based triage test for women whose primary cervical cancer screening results are positive for the human papillomavirus (HPV). 

The test uses the cobas 4800 HPV, which simplifies clinical decision-making by providing simple results, so clinicians and women understand next steps following diagnosis.

“Despite being nearly 100 percent preventable, cervical cancer is still one of the most common cancers in women worldwide. To address this, Roche is dedicated to investing in next-generation biomarkers that will significantly advance screening strategies and support global efforts to eradicate this disease,” Thomas Schinecker, CEO of Roche, said in the announcement.

Although HPV infections are usually treatable, some women still develop pre-cancerous cervical lesions that, if left untreated, may progress for cervical cancer. 

CINtec PLUS Cytology provides information about which HPV-positive women may need, from referrals to colonoscopy versus repeat testing. This system is a step in the right direction to individualize women’s care and prevent overtreatment and undertreatment.