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New FDA Approvals

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Several new drugs have been approved since April of this year including treatments for migraine, liver disease, kidney disease, and those facing opioid withdrawal symptoms.

Aimovig (erenumab-aooe) from Amgen has been approved for the preventive treatment of episodic or chronic migraine in adults. Aimovig is supplied as an injection with a recommended dosage of 70 mg once per month. Some patients may benefit from two injections of 70 mg once per month. If a dose of Aimovig is missed, another dose should be administered as soon as possible.

Adverse reactions associated with the use of Aimovig may include, but are not limited to, the following:

  • injection site reactions
  • constipation

For those with a rare, inherited, disorder known as X-linked hypophosphatemia (XLH) Crysvita (burosumab-twza) from Ultragenyx has recently been approved.

XLH is characterized by low levels of phosphate in the blood. Phosphate levels are low because phosphate is abnormally processed in the kidneys, which causes a loss of phosphate in the urine and leads to soft, weak bones (rickets).This genetic disorder affects about one in 20,000 people and is typically, it is passed from one generation to the next, but sometimes appears in an individual with no family history of XLH.

Crysvita is specifically indicated for the treatment of X-linked hypophosphatemia (XLH) in adult and pediatric patients one year of age and older and is supplied as an injection every two to four weeks depending on the age of the patient and the length of treatment.

Side effect may include, but are not limited to:

Pediatrics:

  • headache
  • injection site reaction
  • vomiting
  • pyrexia
  • pain in extremity
  • vitamin D decreased

Adults:

  • back pain
  • headache
  • tooth infection
  • restless leg syndrome
  • vitamin D decreased
  • dizziness
  • constipation
  • blood phosphorus increased

AndexXa (coagulation factor Xa (recombinant), inactivated-zhzo) by Portola Pharmaceuticals has recently been approved for the reversal of factor Xa inhibitors. AndexXa is administered via intravenous injection.

This is the first and only antidote indicated for patients treated with drugs such as rivaroxaban and apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding. Factor Xa inhibitors are anticoagulants used to prevent deep vein thrombosis and pulmonary embolism in patients undergoing hip or knee replacement surgery.

Side effects include:

  • urinary tract infections
  • pneumonia

Two new drugs have been approved for the treatment of thrombocytopenia (low blood platelet count).

Doptelet (avatrombopag) from AkaRx has been approved for the treatment of thrombocytopenia in adults with chronic liver disease who are scheduled to undergo a procedure that would typically require platelet transfusion. Doptelet is supplied as a tablet for oral administration with dosing beginning 10-13 days prior to the scheduled procedure.

Side effects may include, but are not limited to:

  • pyrexia
  • abdominal pain
  • nausea
  • headache
  • fatigue
  • edema peripheral

Tavalisse (fostamatinib disodium hexahydrate) from Rigel Pharmaceuticals has been approved for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who have had an insufficient response to a previous treatment.

Tavalisse (fostamatinib disodium hexahydrate) is a tyrosine kinase inhibitor with demonstrated activity against spleen tyrosine kinase (SYK).

Tavalisse is supplied as a tablet for oral administration with an initial dose of 100 mg taken orally twice daily for a month. Depending on platelet county, dosage may be increased. Tavalise may be taken with or without food.

Side effects may include:

  • diarrhea
  • hypertension
  • nausea
  • respiratory infection
  • dizziness
  • ALT/AST increased, rash
  • abdominal pain
  • fatigue
  • chest pain
  • neutropenia

For those with higher than normal potassium levels—known as hyperkalemia—Lokelma (sodium zirconium cyclosilicate) from AstraZeneca has been approved. Lokelma is a highly-selective, oral potassium-removing agent.

Lokelma is administered orally as a suspension in water for oral administration. The recommended dose is 10 g administered three times a day for up to 48 hours. For continued treatment, the recommended dose is 10 g once daily. The most common adverse reaction associated with the use of Lokelma is mild to moderate edema.

Palynziq (pegvaliase-pqpz) from BioMarin has been approved for the treatment of phenylketonuria. Phenylketonuria (commonly known as PKU) is an inherited disorder that increases the levels of a substance called phenylalanine in the blood. Phenylalanine is a building block of proteins (an amino acid) that is obtained through the diet. It is found in all proteins and in some artificial sweeteners.Without treatment, phenylalanine builds up in the blood and causes health problems.

Playnziq is specifically indicated to reduce blood phenylalanine concentrations in adult patients with phenylketonuria who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.

Side effects of Palynziq may include:

  • injection site reactions
  • arthralgia
  • hypersensitivity reactions
  • headache
  • generalized skin reactions lasting at least 14 days
  • pruritus
  • nausea
  • abdominal pain
  • oropharyngeal pain
  • vomiting
  • cough
  • diarrhea
  • fatigue

Jynarque (tolvaptan) by Otsuka America has been approved for the treatment of autosomal dominant polycystic kidney disease (PKD). PKD is a genetic disorder characterized by the growth of numerous cysts filled with fluid in both kidneys. The progressive expansion of PKD cysts slowly replaces much of the normal mass of the kidneys, and can reduce kidney function and lead to kidney failure.

Jynarque is a selective vasopressin V2-receptor antagonist that is specifically indicated to slow kidney function decline in adults at risk of rapidly progressing autosomal dominant polycystic kidney disease.

Jynarque is supplied as a tablet for oral administration. The recommended initial dose is 60 mg orally per day as 45 mg taken on waking and 15 mg taken 8 hours later. Titrate to 60 mg plus 30 mg then to 90 mg plus 30 mg per day if tolerated with at least weekly intervals between titrations. Patients may down-titrate based on tolerability.

Adverse effects associated with the use of Jynarque may include, but are not limited to:

  • thirst
  • polyuria
  • nocturia
  • pollakiuria
  • polydipsia

Jynarque (tolvaptan) is not without risk and can cause serious and potentially fatal liver injury. Acute liver failure requiring liver transplantation has been reported.

Lucemyra(lofexidine) by US Worldmeds has been approved for the mitigation of opioid symptoms to facilitate abrupt opioid discontinuation in adults.

Lucemyra is supplied as a tablet for oral administration. The usual starting dosage is three 0.18 mg tablets taken orally 4 times daily during the period of peak withdrawal symptoms (generally the first 5 to 7 days following last use of opioid) with dosing guided by symptoms and side effects. There should be 5 to 6 hours between each dose. The total daily dosage of Lucemyra should not exceed 2.88 mg (16 tablets) and no single dose should exceed 0.72 mg (4 tablets).

Lucemyra treatment may be continued for up to 14 days with dosing guided by symptoms.  Discontinue Lucemyra with a gradual dose reduction over a 2- to 4-day period to mitigate Lucemyra withdrawal symptoms.

Adverse effects associated with the use of Lucemyra may include, but are not limited to:

  • orthostatic hypotension
  • bradycardia
  • hypotension
  • dizziness
  • somnolence
  • sedation
  • dry mouth

Lucemyra may cause low blood pressure or slower heart rate and should be used with caution in patients diagnosed with low blood pressure, slow heart rate, any other cardiac abnormality (including prior diagnosis or family history of long QT syndrome), or prior heart attack.

 

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New FDA Approvals

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A few new drugs have been approved by the FDA so far this year. Among them are treatments for cystic fibrosis, prostate cancer, pancreatic cancer, and two new treatments for HIV.
Symdeko from Vertex is a combination of tezacaftor and ivacaftor for the treatment of cystic fibrosis in patients age 12 years and older. Tezacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) corrector. Ivacaftor is a CFTR potentiator.
Symdeko is specifically indicated for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.
The recommended dose of Symdeko is one tablet (tezacaftor 100 mg/ivacaftor 150 mg) taken in the morning and one tablet (ivacaftor 150 mg) taken in the evening, approximately 12 hours apart. The tablets should be swallowed whole. Symdeko should be taken with fat-containing food.
Adverse effects associated with the use of Symdeko may include, but are not limited to:

  • headache
  • nausea
  • sinus congestion
  • dizziness

Erleada from Janssen Oncology was approved in February to treat a certain type of prostate cancer using novel clinical trial endpoint.  Erleada works by blocking the effect of androgens, a type of hormone, on the tumor. These androgens, such as testosterone, can promote tumor growth.
“The FDA evaluates a variety of methods that measure a drug’s effect, called endpoints, in the approval of oncology drugs. This approval is the first to use the endpoint of metastasis-free survival, measuring the length of time that tumors did not spread to other parts of the body or that death occurred after starting treatment,” said Richard Pazdur, M.D., director of the 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 the trial supporting approval, Erleada had a robust effect on this endpoint. This demonstrates the agency’s commitment to using novel endpoints to expedite important therapies to the American public.”
Adverse effects associated with the use of Erleada may include:

  • fatigue
  • hypertension
  • rash
  • diarrhea
  • nausea
  • weight decreased
  • arthralgia
  • fall
  • hot flush
  • decreased appetite
  • fracture
  • peripheral edema

Advanced Accelerator Applications has recently received FDA approval for Lutathera to treat a type of cancer that affects the pancreas or gastrointestinal tract called gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Lutathera (lutetium Lu 177 dotatate) is a radiolabeled somatostatin analog.

Lutathera is supplied as an injection for intravenous administration. The recommended dose is 7.4 GBq (200 mCi) every 8 weeks for a total of 4 doses.
Adverse effects may include, but are not limited to, the following:

  • lymphopenia
  • increased GGT
  • vomiting
  • nausea
  • increased AST
  • increased ALT
  • hyperglycemia
  • hypokalemia

Biktarvy from Gilead was approved in February for the treatment of HIV-1 infection in adults.
Biktarvy is specifically indicated as a complete regimen for the treatment of HIV-1 infection in adults who have no antiretroviral treatment history or to replace the current antiretroviral regimen in those who are virologically suppressed (HIV-1 RNA <50 c/mL) on a stable antiretroviral regimen for at least three months with no history of treatment failure and no known substitutions associated with resistance to the individual components of Biktarvy.
Biktarvy is supplied as a tablet for oral administration. The recommended dose is one tablet once daily with or without food.
Adverse events associated with the use of Biktarvy may include:

  • diarrhea
  • nausea
  • headache

TaiMed Biologics has received FDA approval for Trogarzo to treat HIV patients who have tried multiple HIV medications in the past and whose HIV infections cannot be successfully treated with other currently available therapies (multidrug resistant HIV, or MDR HIV). This antiretroviral medication is administered once every 14 days and used in combination with other limited antiretroviral medications.
“While most patients living with HIV can be successfully treated using a combination of two or more antiretroviral drugs, a small percentage of patients who have taken many HIV drugs in the past have multidrug resistant HIV, limiting their treatment options and putting them at a high risk of HIV-related complications and progression to death,” said Jeff Murray, M.D., deputy director of the Division of Antiviral Products in the FDA’s Center for Drug Evaluation and Research. “Trogarzo is the first drug in a new class of antiretroviral medications that can provide significant benefit to patients who have run out of HIV treatment options. New treatment options may be able to improve their outcomes.”
Adverse effects associated with the use of Trogarzo may include:

  • diarrhea
  • dizziness
  • nausea
  • rash

 

Final drug approvals from 2016

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The FDA has approved a few more new medications to close out 2016.
Spinraza was just recently approved to treat children and adults with spinal muscular atrophy (SMA), a rare and often fatal genetic disease affecting muscle strength and movement. Spinraza is an injection administered into the fluid surrounding the spinal cord.

“There has been a long-standing need for a treatment for spinal muscular atrophy, the most common genetic cause of death in infants, and a disease that can affect people at any stage of life,” said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA’s Center for Drug Evaluation and Research. “We worked hard to review this application quickly; we could not be more pleased to have the first approved treatment for this debilitating disease.”
SMA is a hereditary disease that causes weakness and muscle wasting because of the loss of lower motor neurons controlling movement. There is wide variability in age of onset, symptoms and rate of progression. Spinraza is approved for use across the range of spinal muscular atrophy patients.
Rubraca was also approved in December to treat women with a advanced ovarian cancer who have been treated with two or more chemotherapies and whose tumors have a specific gene mutation (deleterious BRCA) as identified by an FDA-approved companion diagnostic test.
“Today’s approval is another example of the trend we are seeing in developing targeted agents to treat cancers caused by specific mutations in a patient’s genes,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and acting director of the FDA’s Oncology Center of Excellence.
“Women with these gene abnormalities who have tried at least two chemotherapy treatments for their ovarian cancer now have an additional treatment option.”
The National Cancer Institute estimates that 22,280 women will be diagnosed with ovarian cancer in 2016 and an estimated 14,240 will die of this disease. Approximately 15 to 20 percent of patients with ovarian cancer have a BRCA gene mutation.
For patients two years of age and older with mild to moderate eczema, Eucrisa was approved. Atopic dermatitis, a chronic inflammatory skin disease, is often referred to as “eczema,” which is a general term for the several types of inflammation of the skin. Atopic dermatitis is the most common of the many types of eczema and onset typically begins in childhood and can last through adulthood. The cause of atopic dermatitis is a combination of genetic, immune and environmental factors. In atopic dermatitis, the skin develops red, scaly and crusted bumps, which are extremely itchy. Scratching leads to swelling, cracking, “weeping” clear fluid, and finally, coarsening and thickening of the skin.
“Today’s approval provides another treatment option for patients dealing with mild to moderate atopic dermatitis,” said Amy Egan, deputy director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research (CDER).
Serious side effects of Eucrisa include hypersensitivity reactions. Eucrisa should not be used in patients who have had a hypersensitivity reaction to Eucrisa’s active ingredient, crisaborole. The most common side effect of Eucrisa is application site pain, including burning or stinging.
Zinplava was approved in October to reduce the recurrence of Clostridium difficile infection (CDI) in patients aged 18 and older. Zinplava is used together with antibacterial drugs that are given to treat CDI. Clostridium difficile causes inflammation of the colon and diarrhea, which can be deadly. Zinplava is administered intravenously by healthcare professionals.
Adverse effects associated with the use of Zinplava may include, but are not limited to, the following:

  • nausea
  • pyrexia
  • Headache

In addition, heart failure was reported more commonly in Zinplava-treated patients with a history of congestive heart failure (CHF) in the two Phase III clinical trials. In patients with a history of CHF, Zinplava should be reserved for use when the benefit outweighs the risk.
The treatment of soft tissue sarcoma just got some help with the approval of Lartruvo with doxorubicin to treat adults with certain types of soft tissue sarcoma (STS), which are cancers that develop in muscles, fat, tendons or other soft tissues. Lartruvo is approved for use with the FDA-approved chemotherapy drug doxorubicin for the treatment of patients with STS who cannot be cured with radiation or surgery and who have a type of STS for which an anthracycline (chemotherapy) is an appropriate treatment.
“For these patients, Lartruvo, added to doxorubicin, provides a new treatment option,” said Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research and acting director of the FDA’s Oncology Center of Excellence. “This is the first new therapy approved by the FDA for the initial treatment of soft tissue sarcoma since doxorubicin’s approval more than 40 years ago.”
And finally, Exondys 51 was just approved to treat patients with Duchenne muscular dystrophy. This is the first drug approved to treat patients with Duchenne muscular dystrophy (DMD). Exondys 51 is specifically indicated for patients who have a confirmed mutation of the dystrophin gene amenable to exon 51 skipping, which affects about 13 percent of the population with DMD.
“Patients with a particular type of Duchenne muscular dystrophy will now have access to an approved treatment for this rare and devastating disease,” said Janet Woodcock, M.D., director of the FDA’s Center for Drug Evaluation and Research. “In rare diseases, new drug development is especially challenging due to the small numbers of people affected by each disease and the lack of medical understanding of many disorders. Accelerated approval makes this drug available to patients based on initial data, but we eagerly await learning more about the efficacy of this drug through a confirmatory clinical trial that the company must conduct after approval.”
DMD is a rare genetic disorder characterized by progressive muscle deterioration and weakness. It is the most common type of muscular dystrophy. DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact.The first symptoms are usually seen between three and five years of age, and worsen over time. The disease often occurs in people without a known family history of the condition and primarily affects boys, but in rare cases it can affect girls. DMD occurs in about one out of every 3,600 male infants worldwide.
People with DMD progressively lose the ability to perform activities independently and often require use of a wheelchair by their early teens. As the disease progresses, life-threatening heart and respiratory conditions can occur. Patients typically succumb to the disease in their 20s or 30s; however, disease severity and life expectancy vary.
 
Source: FDA
 

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