Top 3 new FDA approvals

October 1, 2018

Here are the top 3 new approvals from FDA.

In late September, FDA approved a first-of-its-kind cancer test, as well as the first advanced carcinoma treatment and a drug to treat a lung disease caused by a group of bacteria.

Here are the top 3 new FDA approvals:

1. ClonoSEQ assay (Adaptive Biotechnologies) is a novel, next generation sequencing (NGS)-based test for minimal residual disease (MRD) in patients with acute lymphoblastic leukemia (ALL) or multiple myeloma. MRD measures the amount of cancer cells remaining in a person’s bone marrow.

The in vitro diagnostic uses multiplex polymerase chain reaction (PCR) and NGS to identify and quantify certain gene sequences in DNA extracted from bone marrow from patients with ALL or multiple myeloma. Plus, the ClonoSEQ assay is capable of detecting MRD at levels below 1 in 1 million cells, FDA said in a statement.

The highly sensitive test can help providers manage their patients’ care, said FDA Commissioner Scott Gottlieb in the statement. “Today’s approval is an important step forward for patients suffering from ALL and multiple myeloma. Determining whether a patient has residual cancer cells remaining after treatment provides information on how well a patient has responded to therapy and how long remission may last.”

FDA is applying novel regulatory approaches to ensure that the rapidly-evolving NGS tests are accurate and reliable, Gottlieb added. “At the same time, we’re seeing more and more laboratory-developed tests seek marketing authorization from the FDA. We’re doing as much as we can to advance these opportunities for patients under our current authorities. But we believe that to more fully unlock these innovations, we need to modernize the regulatory framework for all in vitro clinical tests.”

Related: FDA clears new type of leukemia treatment

2. Cemiplimab-rwlc (Libtayo, Regeneron Pharmaceuticals) injection for IV use is the first FDA approval of a drug specifically for advanced cutaneous squamous cell carcinoma (CSCC).

Libtayo treats patients with metastatic CSCC or locally advanced CSCC who are not candidates for curative surgery or curative radiation.

“We’re continuing to see a shift in oncology toward identifying and developing drugs aimed at a specific molecular target. With the Libtayo approval, the FDA has approved six immune checkpoint inhibitors targeting the the PD-1 / PD-L1 pathway for treating a variety of tumors, from bladder to head and neck cancer, and now advanced CSCC,” said Richard Pazdur, MD, director of FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a statement from the agency. “This type of cancer can be difficult to treat effectively when it is advanced and it is important that we continue to bring new treatment options to patients.”

CSCC is the second most common human cancer in the United States with an estimated annual incidence of approximately 700,000 cases.

Related: Breast cancer drug first to get nod in FDA accelerated approval program

3. Amikacin liposome inhalation suspension (Arikayce, Insmed) treats a lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC), in a limited population of patients with the disease who do not respond to conventional treatment (refractory disease).
MAC is a type of nontuberculous mycobacteria (NTM) commonly found in water and soil. Symptoms of disease in patients with MAC include persistent cough, fatigue, weight loss, night sweats, and occasionally shortness of breath and coughing up of blood.

"As bacteria continue to grow impervious to currently available antibiotics, we need to encourage the development of drugs that can treat resistant infections. That means utilizing novel tools intended to streamline development and encourage investment into these important endeavors," said FDA Commissioner Scott Gottlieb, MD, in a statement from the agency.

Arikayce is the first treatment approved under the Limited Population Pathway for Antibacterial and Antifungal Drugs. “This pathway, advanced by Congress, aims to spur development of drugs targeting infections that lack effective therapies. We’re seeing a lot of early interest among sponsors in using this new pathway, and it’s our hope that it’ll spur more development and approval of antibacterial drugs for treating serious or life-threatening infections in limited populations of patients with unmet medical needs,” Gottlieb said.

Arikayce’s prescribing information includes a Boxed Warning regarding the increased risk of respiratory conditions including hypersensitivity pneumonitis (inflamed lungs), bronchospasm (tightening of the airway), exacerbation of underlying lung disease and hemoptysis (spitting up blood) that have led to hospitalizations in some cases.

Read more: FDA okays radiation drug for ultra-rare cancers