New FDA Approval

The FDA has recently approved more than a dozen new medications for conditions ranging from sickle cell anemia to Parkinson’s disease.
Actemra (tocilizumab) injection from Genentech was approved in September for the treatment of CAR T cell-induced severe or life-threatening cytokine release syndrome. Actemra is specifically indicated for adults and pediatric patients 2 years of age and older with chimeric antigen receptor (CAR) T cell-induced severe or life-threatening cytokine release syndrome.
Side effects may include:

• upper respiratory tract infections
• nasopharyngitis
• headache
• hypertension
• increased ALT
• injection site reactions

Bayer introduces Aliqopa (copanlisib) for the treatment of follicular lymphoma. Aliqopa was approved in September and is specifically indicated for the treatment of adult patients with relapsed follicular lymphoma (FL) who have received at least two prior systemic therapies.
Side effects may include:

• hyperglycemia
• diarrhea
• decreased general strength and energy
• hypertension
• leukopenia
• neutropenia
• nausea
• lower respiratory tract infections
• thrombocytopenia

Benznidazole from the Chemo Group was approved in August for the treatment of Chagas disease. Benznidazole is specifically indicated for use in pediatric patients 2 to 12 years of age for the treatment of Chagas disease (American trypanosomiasis), caused by Trypanosoma cruzi. According to the CDC, Chagas disease is considered one of the neglected parasitic infections (NPI), a group of five parasitic diseases that have been targeted by CDC for public health action in the United States. Some side effects include:

• stomach pain
• rash
• decreased weight
• headache
• nausea
• vomiting
• abnormal white blood cell count

For the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia Pfizer introduced Besponsa (inotuzumab ozogamicin) and it was approved in August.
Besponsa is specifically indicated for the treatment of adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia. Some side effects include:

• infection
• anemia
• fatigue
• hemorrhage
• pyrexia
• nausea
• headache

Duzallo (lesinurad and allopurinol) from Ardea Biosciences was approved in August for the treatment of hyperuricemia associated with gout. Duzallo is specifically indicated for the treatment of hyperuricemia associated with gout in patients who have not achieved target serum uric acid levels with a medically appropriate daily dose of allopurinol alone. Side effects may include:

• headache
• influenza
• blood creatinine increased
• gastroesophageal reflux disease
• skin rash

Another treatment for sickle cell disease has been approved. Endari (L-glutamine oral powder) from Emmaus Life Sciences was approved in July 2017 to reduce the severe complications of sickle cell disease (SCD) in adult and pediatric patients age 5 and older. Side effects may include:

• constipation
• nausea
• headache
• abdominal pain
• cough
• pain in extremity
• back pain
• chest pain (non-cardiac)

For patients with Parkinson’s disease dyskinesia, Gocovri (amantadine) from Adamas Pharmaceuticals was approved in August. Gocovri is specifically indicated for the treatment of dyskinesia in patients with Parkinson’s disease receiving levodopa-based therapy, with or without concomitant dopaminergic medications. Adverse effects may include, but are not limited to:

• hallucination
• dizziness
• dry mouth
• peripheral edema
• constipation
• fall
• orthostatic hypotension

IDHIFA (enasidenib) from Celgene was approved in August for the treatment of relapsed or refractory acute myeloid leukemia with IDH2 mutation. IDHIFA is specifically indicated for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (AML) with an isocitrate dehydrogenase-2 (IDH2) mutation as detected by an FDA-approved test. Side effects may include:

• nausea
• vomiting
• diarrhea
• increased levels of bilirubin
• decreased appetite

KedRab [Rabies Immune Globulin (Human)] from Kedrion Biopharma was recently approved for the post-exposure prophylaxis of rabies infection. KedRab is specifically indicated for passive, transient post-exposure prophylaxis of rabies infection, when given immediately after contact with a rabid or possibly rabid animal. Side effects may include:

• injection site pain
• headache
• muscle pain
• upper respiratory tract infection

Kymriah (tisagenlecleucel) from Novartis was approved in August for the treatment of refractory B-cell precursor acute lymphoblastic leukemia. Kymriah is specifically indicated for the treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse. Some side effects include:

• cytokine release syndrome
• pyrexia
• decreased appetite
• headache
• encephalopathy
• hypotension
• bleeding episodes
• tachycardia
• nausea
• diarrhea
• vomiting
• viral infectious disorders
• fatigue
• acute kidney injury

Mavyret (glecaprevir and pibrentasvir) by AbbVie was approved for the treatment of adult patients with chronic hepatitis C virus (HCV) genotype 1, 2, 3, 4, 5 or 6 infection without cirrhosis or with compensated cirrhosis (Child-Pugh A). Mavyret is also indicated for the treatment of adult patients with HCV genotype 1 infection, who previously have been treated with a regimen containing an HCV NS5A inhibitor or an NS3/4A protease inhibitor (PI), but not both. Side effects include:

• headache
• fatigue

Solosec (secnidazole) by Symbiomix Therapeutics was recently approved for the treatment of bacterial vaginosis in adult women. Side effects may include:

• vulvo-vaginal candidiasis
• headache
• nausea
• dysgeusia
• vomiting
• diarrhea
• abdominal pain
• vulvovaginal pruritus

For adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy, Tremfya (guselkumab) from Janssen Biotech has recently been approved. Side effects could include:

• upper respiratory infections
• headache
• injection site reactions
• arthralgia
• diarrhea
• gastroenteritis
• tinea infections
• herpes simplex infections

Vabomere (meropenem and vaborbactam) from The Medicines Company has been approved for the treatment of adults with complicated urinary tract infections, including pyelonephritis caused by designated susceptible bacteria. Side effect may include:

• headache
• phlebitis/infusion site reactions
• diarrhea

Verzenio (abemaciclib) from Eli Lilly was recently approved for the treatment of HR+, HER2- breast cancer. Verzenio is specifically indicated for use:

• in combination with fulvestrant for the treatment of women with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative advanced or metastatic breast cancer with disease progression following endocrine therapy;
• as monotherapy for the treatment of adult patients with HR-positive, HER2-negative advanced or metastatic breast cancer with disease progression following endocrine therapy and prior chemotherapy in the metastatic setting. 

Side effects may include:

• diarrhea
• neutropenia and leukopenia
• nausea
• abdominal pain
• infections
• fatigue
• anemia
• decreased appetite
• vomiting
• headache

For adult patients with newly-diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes, Vyxeos (daunorubicin and cytarabine) from Jazz Pharma has been approved. Side effects may include, but are not limited to:

• hemorrhagic events
• febrile neutropenia
• rash
• edema
• nausea
• mucositis
• diarrhea
• constipation
• musculoskeletal pain
• fatigue
• abdominal pain
• dyspnea
• headache
• cough
• decreased appetite
• arrhythmia
• pneumonia
• bacteremia
• chills
• sleep disorders
• vomiting