Brineura/cerliponase alfa Cost, Side effects, Dosage, Uses for Batten disease

Brineura/cerliponase alfa Cost, Side effects, Dosage, Uses for Batten disease. The FDA granted approval of Brineura to BioMarin Pharmaceutical Inc

US FDA approved new medicine Brineura that contains active pharmaceutical ingredient cerliponase alfa for the treatment of specific type of batten disease. This first drug approved by US FDA to reduce the severity of symptoms like impaired walking ability in pediatric patients having age less than 3 years or more suffering from late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.

Batten disease is extremely rare kind of disease and characterized by fatal autosomal recessive neuro-degenerative disorder. It is kind of neuro-degenerative disorder and placed in a group of disorders termed as  neuronal ceroid lipofuscinoses (NCLs). So far 20 genes have been identified that have linked with batten disease out of which type 3 (CLN3) has high incidence rate.

Major sign and symptoms of batten disease include vision difficulties, mood changes, variation in behavior, mental retardation, speech difficulties, seizures and poor circulation of blood in extremities. It has been observed that in females symptoms develops later than in males but their survival life span is shorter than males. Batten disease is simply a genetic disorder so, people having batten disease in their family history are more prone to batten disease.

Brineura/cerliponase alfa Cost, Side effects, Dosage, Uses for Batten disease


Brineura/cerliponase alfa Side effects

According to data provided by US FDA drug exhibit following side effects during clinical trials
  • Changes in ECG waves
  • Decreased heart rate
  • Anaphylaxis
  • Nausea 
  • Vomiting
  • Seizures
  • hemorrhage
  • Rash
  • Increase in CSF protein
  • Infection at injection  site

Brineura/cerliponase alfa Uses or Patient Information


This first drug approved by US FDA  for the treatment of specific form of batten and to slow down the loss of walking ability (ambulation) in symptomatic pediatric patients 3 years of age and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency.

Brineura/cerliponase alfa Dosage


This drug is formulated as intraventricular injection administered via intraventricular administration kit. This kit includes

  • Two 20-mL syringes
  • Two syringe needles (21 ga, 25.4 mm)
  • One extension line
  • One infusion set with 0.2 micron inline filter
  • One port needle (22 ga, 16 mm)

Standard dosage frequency of Brineura is 300mg by intraventricular infusion once every other week in patients having age 3 years or more. Drug Safety profile has not yet evaluated in patents having age less than 3 years.

Brineura/cerliponase alfa Cost/Price


It is assuming that expected cost of Brineura per carton will be approximately $27,000 , with patients requiring one carton every other week, according to BioMarin execs.

Rydapt/midostaurin Cost, Side effects, Dosage, Uses for acute myeloid leukemia (AML)

Rydapt/midostaurin Cost, Side effects, Dosage, Uses for acute myeloid leukemia (AML) The FDA granted the approval of Rydapt to Novartis Pharmaceuticals Corporation. 

US FDA approved new medicine Rydapt that contains midostaurin as an active pharmaceutical ingredient for the treatment of acute myeloid leukemia in those adults who have fms like tyrosine kinase 3 (FLT-3) mutation. FDA indicated the usage of this drug along LeukoStrat CDx FLT3 ( Mutation detection Assay used to detect the FLT3 mutation in patients with AML).

Acute myeloid leukemia (AML) is a type of cancer of myeloid cells characterized by instant proliferation of abnormal white blood cells (WBC's). Mortatlity rate of Acute myeloid leukemia (AML) is 1.2% of cancer deaths in the United States. Though, it is rare disease but middle age group is most commonly affected by AML. Acute myeloid leukemia (AML) has several types It is presumed that regular exposure to chemical agents aromatic compounds like benzene, ionizing agents, radicals, halogens and oxidative agents are major risk factors that play important role in pathogenesis of Acute myeloid leukemia (AML). Other risk factors include genetic predisposition, blood disorders, viral infections etc.

Symptoms of Acute myeloid leukemia (AML) include flu like symptoms, restlessness, mild fever, weight loss, loss of appetite, gums swelling, enlarged spleen, internal hemorrhage, petechiae, frequent infections. In some cases Lymph node swelling may also occur but is rare. Patient having age less than 50 years have more survival rate than those having age more than 60 years. 


Rydapt/midostaurin Cost, Side effects, Dosage, Uses for acute myeloid leukemia (AML)

Rydapt/midostaurin Side effects


According to data provided by US FDA this drug exhibit following side effects during clinical trials


  • Drug induced neutropenia
  • Nausea 
  • Vomiting
  • Mucous membrane swelling
  • Diarrhea
  • fatigue
  • abdominal pain
  • Constipation
  • Fever
  • muscular pain
  • shortness of breadth
  • Upper respiratory tract infections
  • Asthenia
  • Loss of appetite

Rydapt/midostaurin Uses or Patient Information


US FDA assigned Rydapt as breakthrough therapy for the treatment of acute myeloid leukemia (AML) in those patients in which fms like tyrosine kinase 3 (FLT-3) mutations have identified. This drug can be used with other chemotherapeutic agents. Additionally, US FDA also indicated the usage of this drug with LeukoStrat CDx FLT3 ( Mutation detection Assay used to detect the FLT3 mutation in patients with AML).

Rydapt/midostaurin Dosage



Rydapt/midostaurin is formulated  as 25mg tablet for oral administration. It is dosage frequency is 50mg orally (PO) twice a day (BID). This drug may also be used in combination with other chemotherapeutic agents like cytarabine and daunorubicin.

Rydapt/midostaurin Cost/Price

Rydapt/midostaurin is now available in market. Each pack of Rydapt/midostaurin contains 28 capsules (28's) with potency of 25mg. Approximate cost of each pack of Rydapt/midostaurin is 3800$ to 4000$. This price may variate with the passage of time so, make it confirm by visiting your nearby pharmacy before purchasing.

Ingrezza/Valbenazine Cost, Side effects, Dosage, Uses for Tardive Dyskinesia

Ingrezza/Valbenazine Cost, Side effects, Dosage, Uses for Tardive Dyskinesia. The FDA granted approval of Ingrezza to Neurocrine Biosciences, Inc.

US FDA approved new drug Ingrezza that contains valbenazine as an active pharmaceutical ingredient for the treatment of tardive dyskinesia in adults. This first kind of therapy approved by US FDA as a licensed treatment for tardive dyskinesia. This drug belongs to therapeutic class termed as  vesicular monoamine transporter 2 (VMAT2) inhibitors.

Tardive dyskinesia (TD) is a severe and incurable form of dyskinesia characterized by repetitive involuntary body movements. It is termed as tardive because movements in this type of disorder are slow or belated onset. One of the major cause of this disorder is abusive use of anti psychotic drugs for a long term.

Exact underlying mechanism is still uncleared to somehow but many researcher and health care providers proposed over used of anti-psychotic drugs, smoking, neuro-degeneration, genetic predisposition, increase age are those factors that have strong correlation with this disease. Common manifestation of tardive dyskinesia are grimacing, eye blinking, lip smacking, tongue movements and lip puckering. Rapid involuntary movements of extremities may also occur. Respiratory problems like difficult breathing, grunting and respiratory depression may feel by patient suffering
tardive dyskinesia.

Ingrezza/Valbenazine is VMAT-2 inhibitor.It works by reducing the dopamine release into synaptic cleft by inhibiting VMAT2 at pre-synaptic area. VMAT2 are responsible for dopamine loading into synaptic vesicles to release them into synaptic cleft. Thus inhibition of VMAT2 creates reversible reduction of dopamine in synaptic cleft.


IngrezzaValbenazine Cost, Side effects, Dosage, Uses for Tardive Dyskinesia




Ingrezza/Valbenazine Side Effects


According to data provided by health authorities drug exhibit moderate to severe kind of side effects during clinical trials. These side effects were

  • Sleepiness 
  • Palpitation
  • Nervousness
  • Headache
  • Prolongation of QT interval
  • Cardiac Arrhythmia
  • Mental  


Ingrezza/Valbenazine Uses/Patient Information


This is the first drug approved by US FDA  (licensed treatment) for the treatment of tardive dyskinesia in adults. In pediatric patients safety profile of this drug has not yet been evaluated.


Ingrezza/Valbenazine Dosage


Standard dosage frequency of this drug is 40mg once a day but in some cases dosage frequency may increased up-to  80mg once a day orally after first week of therapy. Dose adjustment should be considered in case of liver or hepatic impairment. 


Ingrezza/Valbenazine Cost/Price


Ingrezza 40mg capsules are now available in market. Each of pack of Ingrezza 40mg contains 30 capsules (30's) with an estimated average cost of 5000$ - 6000$. Ingrezza 80mg capsules are not yet avialble in market. This price may variate with the passage of time so, make it confirm by visiting your nearby pharmacy before purchasing.