FDA Approves New Opioid For Intravenous Use in Hospitals

FDA recently approved Trevena Inc.’s Olinvyk (oliceridine), an opioid used to manage severe acute pain in adults who require an intravenous opioid and who have been unresponsive to alternative treatments. 

The opioid should be used for short-term intravenous use in hospitals or other controlled clinical settings, including during inpatient and outpatient procedures.

 It is not indicated for at-home use, FDA said. 

So far, 1,535 patients with moderate to severe acute pain have been treated with Olinvyk in open-label trials, and they reported a decreased pain compared to a placebo.

The safety profile for Olinvyk is similar to other opioids, but FDA stated that it should not be given to patients with respiratory depression, acute or severe bronchial asthma, gastrointestinal obstruction, or known hypersensitivity.

“We will continue to do everything we can to reduce the number of Americans who are addicted to opioids and cut the rate of new addiction through a number of cross-agency initiatives,” Douglas Throckmorton MD, deputy director for regulatory programs in the FDA’s Center for Drug Evaluation and Research, said in the announcement. 

“Importantly, the FDA will only approve new drug applications, including those for opioid medications, following a rigorous review to evaluate the risks and benefits and ultimate determination that the data support safety and effectiveness.”

Oral Treatment for Spinal Muscular Atrophy OK’d by FDA

FDA recently approved Roche’ Evrysdi (risdiplam) for the treatment of patients two months of age or older with spinal muscular atrophy (SMA).

Evrysdi is second drug approved to treat SMA, but the first oral drug approved to help patients with the hereditary condition that causes weakness and muscle wasting

“Evrysdi is the first drug for this disease that can be taken orally, providing an important treatment option for patients with SMA, following the approval of the first treatment for this devastating disease less than four years ago,” Billy Dunn, MD, director of the office of neuroscience in FDA’s Center for Drug Evaluation and Research, said in the announcement.

The FDA approval was based on two clinical studies, the infantile-onset SMA study and a randomized, placebo-controlled study.

The first study included 21 patients with an average age of nearly seven months. Evrysdi was evaluated based on the ability to sit without support for at least five seconds and survival without permanent ventilation. 

After 12 months of treatment, 41 percent of patients were able to sit independently for more than five seconds.

Additionally, after 23 or more months of treatment, 81 percent of patients were alive without permanent ventilation, researchers said. 

The second study included 180 patients aged two to 25 years. The primary endpoint was the change from baseline to the one-year mark.

In this study, patients showed an average 1.36 increase in their score at one year, compared to a 0.19 decrease in patients on placebo. 

GSK Gets Greenlight for Multiple Myeloma Drug

FDA recently approved GSK’s BLENREP (belantamab mafodotin-blmf) as a monotherapy treatment for adult patients with relapsed or refractory multiple myeloma.

BLENREP is GSK’s fifth medicine approval in 2020 across areas of vital unmet medical needs including cancer, HIV, and chronic kidney disease.

FDA stated that the drug is specifically intended for patients who have previously received at least four therapies, including an anti-CD38 monoclonal antibody, a proteasome inhibitor, and an approved based on response rate.

“As the second most common form of blood cancer in the US, multiple myeloma is an incurable and devastating disease. BLENREP is the first approved anti-BCMA therapy and has the potential to transform the treatment of patients with relapsed or refractory myeloma who have limited treatment options today,” said Hal Barron, MD, GSK’s chief scientific officer and president R&D.

The FDA approval was based on six-month primary results from the pivotal DREAMM-2 study, which enrolled patients with relapsed or refractory multiple myeloma that had worsened despite standard care.

The overall response rate for BLENREP in patients who received a median of seven prior lines of treatment was 31 percent. In addition, 73 percent of responders had duration of response equal to or greater than six months, researchers said.

FDA Approves Liquid Biopsy Sequencing Companion Diagnostic Test

FDA recently approved the first liquid biopsy companion diagnostic to identify patients with a specific type of mutation of the epidermal growth factor receptor gene in a deadly form of metastatic non-small cell lung cancer.

The liquid biopsy, Guardant360, is the first approved test that combines two technologies in one diagnostic test, according to the federal agency.

The first technology leverages liquid biopsy, which uses a blood sample to provide healthcare professionals with genetic information about a patient’s tumor, FDA said.

Liquid biopsies are less invasive than standard tissue biopsies.

The second technology that Guardant360 uses is NGS, which use large-panel genetic sequencing. NGS requires only one test in order to allow clinicians to better assess a tumor composition.

Additionally, NGS technology detects mutations in 55 tumor genes, rather than one gene at a time.

“Approval of a companion diagnostic that uses a liquid biopsy and leverages next-generation sequencing marks a new era for mutation testing,” Tim Stenzel, MD, PhD, director of the office of in vitro diagnostics and radiological health in the FDA’s center for devices and radiological health. 

“In addition to benefitting from less invasive testing, patients are provided with a simultaneous mapping of multiple biomarkers of genomic alterations, rather than one biomarker at a time, which can translate to decreased wait times for starting treatment and provide insight into possible resistance mechanisms.”

Rare Duchenne Muscular Dystrophy Mutation Gets Targeted Treatment

FDA recently granted accelerated approval for Viltepso (viltolarsen), an injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.

DMD is a rare genetic condition that includes progressive muscle deterioration and weakness, FDA said. It is the most common type of muscular dystrophy.

The FDA approval was based on two clinical studies, which showed that dystrophin increased, on average, from 0.6 percent of normal at baseline to 5.9 percent of normal at Week 25.

Therefore, FDA concluded that the “applicant’s data demonstrated an increase in dystrophin production that is reasonably likely to predict clinical benefit in patients with DMD who have a confirmed mutation of the dystrophin gene amenable to exon 53 skipping.”

As part of the approval, FDA is requiring that the company conduct a clinical trial to confirm the drug’s clinical benefit and to assess whether Viltepso improves the time to stand for DMD patients with confirmed mutation.