6 New FDA Approvals to Know from April 2017

Learn about the new products and expanded indications approved by the FDA in April 2017.

1. Austedo

The FDA approved Teva’s deutetrabenazine (Austedo) on April 3, 2017.¹

Indicated for the treatment of chorea associated with Huntington’s disease (HD), Austedo is the first deuterated product to receive the FDA’s nod, as well as the second HD therapy to earn the agency’s approval.

Austedo was approved with a boxed warning alerting patients and providers to an associated risk of depression and suicidal thoughts and behaviors in patients with HD. Additionally, the drug is contraindicated in patients with hepatic impairment, patients taking monoamine oxidase inhibitors (MAOIs) or discontinued MAOI therapy within 14 days, patients taking reserpine or who discontinued reserpine within 20 days, and patients taking tetrabenazine (Xenazine).

Adverse events reported by trial participants treated with Austedo include worsening in mood, cognition, rigidity, and functional capacity. The FDA also noted that the drug may increase a patient’s risk of akathisia, agitation, and restlessness, and could cause parkinsonism in patients with HD.

2. Brineura

The FDA has approved BioMarin Pharmaceutical’s cerliponase alfa (Brineura) on April 27, 2017.²

Brineura, an enzyme replacement therapy, is indicated to slow loss of walking ability in patients aged 3 years and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a rare form of Batten disease. It is the first therapy to receive the FDA’s nod for this purpose.

The recommended dose of the drug in pediatric patients aged 3 years an older is 300 mg administered once every other week via intraventricular infusion, followed by an infusion of electrolytes.

Adverse events associated with the use of Brineura include fever, ECG abnormalities such as slow heart rate, hypersensitivity, decrease or increase in CSF protein, vomiting, seizures, hematoma, headache, irritability, increased CSF white blood cell count, device-related infection, and low blood pressure. The drug should not be administered to patients with signs of acute intraventricular access device-related complications or ventriculoperitoneal shunts.

3. Harvoni and Sovaldi

The FDA expanded the indication if Gilead Sciences’ ledipasvir and sofosbuvir (Harvoni) and sofosubuvir (Sovaldi) on April 7, 2017, approving both drugs for the treatment of hepatitis C virus (HCV) in pediatric patients.³

Harvoni and Sovaldi were previously approved to treat HCV in adults, but the latest approval expands the drugs’ use to include children aged 12 to 17 years. Specifically, Harvoni is indicated to treat patients with HCV genotypes 1, 4, 5, or 6 infection without cirrhosis or with mild cirrhosis, while Sovaldi is to be used in combination with ribavirin to treat those with genotype 2 or 3 HCV infection without cirrhosis or with mild cirrhosis.

With this decision, the drugs have become the first direct-acting antiviral treatments approved for children and adolescents with HCV.

Adverse events associated with the use of either Harvoni or Sovaldi include fatigue and headache.

4. Ingrezza

The FDA approved Neurocrine Biosciences’ valbenazine (Ingrezza) on April 11, 2017.⁴

Ingrezza is the first drug to receive the FDA’s nod for the treatment of tardive dyskinesia, a neurological condition characterized by repetitive involuntary movements.

Adverse events reported by trial patients treated with Ingrezza include sleepiness and heart rhythm problems (QT prolongation). The drug should not be used in patients with congenital long QT syndrome or abnormal heartbeats associated with a prolonged QT interval.

5. Rydapt

The FDA approved Novartis’ midostaurin (Rydapt) on April 28, 2017.⁵

Rydapt is approved, in combination with chemotherapy, for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients with the FLT3 genetic mutation, which is to be detected using Invivoscribe Technologies’ LeukoStrat CDx FLT3 Mutation Assay.

The drug can also be used in adults with rare blood disorders such as aggressive systemic mastocytosis, systemic mastocytosis with associated hematological neoplasm, and mast cell leukemia.

Adverse events associated with the use of Rydapt include low levels of white blood cells with fever, nausea, inflammation of the mucous membranes, vomiting, headaches, spots on the skin due to bleeding, musculoskeletal pain, nosebleeds, device-related infection, high blood sugar, and upper respiratory tract infection.

6. Stivarga

The FDA expanded the indication of Bayer HealthCare Pharmaceuticals’ regorafenib (Stivarga) on April 27, 2017.⁶

Previously approved for the treatment of colorectal cancer and gastrointestinal stromal tumors that are no longer responding to previous treatments, the drug can now be used to treat patients with hepatocellular carcinoma who have been previously treated with the drug sorafenib (Nexavar).

Adverse events reported in patients treated with Stivarga include pain, hand-foot skin reactions, fatigue, diarrhea, decreased appetite, high blood pressure, infection, difficulty speaking, high levels of bilirubin in the blood, fever, inflammation of the mucous membranes, weight loss, rash, and nausea.

References

• Teva announces FDA approval of Austedo (deutetrabenazine) tablets for the treatment of chorea associated with Huntington’s Disease [news release]. Teva’s website. http://www.tevapharm.com/news/teva_announces_fda_approval_of_austedo_deutetrabenazine_tablets_for_the_treatment_of_chorea_associated_with_huntington_s_disease_04_17.aspx. Accessed Apr. 4, 2017.
• FDA approves first treatment for a form of Batten disease [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555613.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed April 27, 2017.
• FDA approves two hepatitis C drugs for pediatric patients [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm551407.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed April 7, 2017.
• FDA approves first drug to treat tardive dyskinesia [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm552418.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed April 12, 2017.
• FDA approves new combination treatment for acute myeloid leukemia [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555778.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed April 28, 2017.
• FDA expands approved use of Stivarga to treat liver cancer [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm555608.htm?source=govdelivery&utm_medium=email&utm_source=govdelivery. Accessed April 27, 2017.