Siliq/brodalumab Cost, Side effects, Dosage, Uses for Psoriasis

Siliq is marketed by Bridgewater, New Jersey-based Valeant Pharmaceuticals.

U.S FDA approved new medicine siliq that contains active drug brodalumab for the treatment of psoriasis in adults. Psoriasis is a common auto-immune disorder in among all populations. Psoriasis might be of different types like plaque psoriasis, guttate psoriasis, erythrodermic psoriasis, inverse psoriasis and postular psoriasis. FDA approved Siliq for moderate to severe plaque psoriasis in adults.

Plaque psoriasis is basically a skin disorder that intense irritation and itching on skin and leaves the skin scaly or flaky along with reddish color appearance of skin. This condition may occur without regard to gender, age or ethnicity. However, it is commonly observed that people who have family history of plaque psoriasis or other are more prone to get involved in such disorder.

Siliq/brodalumab belongs to a group termed as monoclonal antibody. Basic mechanism by which brodalumab targets plaque psoriasis is that it binds to receptor interleukin-17 that generates inflammatory mediators to aggravate plaque psoriasis. Interleukin-17 does not activate after binding to brodalumab making it unable to release inflammatory mediators to make condition worse.

Siliq/brodalumab Cost, Side effects, Dosage, Uses for Psoriasis


Siliq/brodalumab  Side effects

During clinical trials drug exhibit some side effects or adverse effects experience by adult participants. These side effects were moderate to severe e.g
  • Fatigue
  • Diarrhea
  • Joint pain (arthralgia)
  • Headache
  • Injection site reactions
  • Influenza
  • low white blood cell count (neutropenia)
  • Fungal (tinea) infections
  • Throat pain (oropharyngeal pain)
  • Nausea, muscle pain (myalgia)   
  • Suicidal tendency 

Siliq/brodalumab Uses 

U.S FDA approved brodalumab for the treatment of  moderate to severe plaque psoriasis in adults. However, you healthcare provider (HCP) may recommend you a combined therapy in order to make treatment successful and better control over disease management.

Siliq/brodalumab Cost and Dosage

Due to risk of suicidal ideation and behavior, the drug will come up with box warning and might be available through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the Siliq REMS Program. We will update you regarding its unit dose and dosage frequency whenever it is available in pharmacies under this program.

Emflaza/deflazacort Cost, Side effects, Dosage, Uses for Duchenne muscular dystrophy (DMD)

Emflaza is marketed by Marathon Pharmaceuticals of Northbrook, Illinois.

US FDA approved new medicine Emflaza that contains active ingredient deflazacort in oral dosage form for the treatment of Duchenne muscular dystrophy (DMD). Emflaza belongs to a therapeutic class termed as corticosteroids. Emflaza is basically a prodrug which is converted in active metabolite after its metabolism. All corticosteroids works by reducing swelling, inflammation and settling down the exaggerated immune response.

Duchenne muscular dystrophy (DMD) is a rare kind of genetic disorder that causes degeneration of muscles. Due to some genetic defect a protein termed as dystrophin does not produce in the body. The basic physiological role of dystrophin is keeping body muscles intact and healthy.

But dystrophin absence in Duchenne muscular dystrophy (DMD) makes muscle more vulnerable to degradation, degeneration and physical deterioration. Symptoms may first appear in the age of 5 years or earlier. After the onset of disease symptoms may get worst with the passage of time.

In extreme cases duchenne muscular dystrophy (DMD) may cause walking disability, cardiovascular or respiratory muscles collapse. These disabilities may appear at any time without regard to age. According to statistics this disease is more common in boys as compared to girls.

Emflaza/deflazacort Cost, Side effects, Dosage, Uses for Duchenne muscular dystrophy (DMD)

Emflaza/deflazacort Side effects

According to clinical data provided by health authorities Emflaza demonstarted a significant results over placebo. However, side effects were similar to other corticosteroids therapy. Most commonly observed side effects were as follows
  • Weight gain
  • Hair growth (hirsutism)
  • Central obesity
  • Facial puffiness (Cushingoid appearance)
  • Upper respiratory tract infection
  • Increased appetite
  • Urinary frequency
  • Cough 

Emflaza/deflazacort Uses/Prescribing information 

Though, number of corticosteroids are being used to muscular disorders like duchenne muscular dystrophy (DMD) But US FDA specifically approved Emflaza in oral dosage form for the treatment of  duchenne muscular dystrophy (DMD) in patients age 5 years and older.

 

Emflaza/deflazacort Dosage

According to medical literature provided this drug will come in following potencies 6mg, 18mg, 30mg and 36mg. Oral suspension of this drug will come up with potency of 22.75mg/mL. Drug will be administered according to body weight by following the standard of 0.9 mg/kg/day orally.

 

Emflaza/deflazacort Cost and Dosage

According to market survey this drug is currently not available at retail pharmacies because it is recently approved by FDA and is under manufacturing process. Once, it will be available at retail outlets we will let you know about the unit cost of drug and its dosage frequency.

Trulance/Plecanatide Cost, Side effects, Dosage, Uses for Chronic Idiopathic Constipation (CIC)

Trulance is manufactured by New York, New York-based Synergy Pharmaceuticals Inc.

U.S FDA approved new drug Trulance that contains active ingredient plecanatide for the treatment of chronic idiopathic constipation in adults. Trulance/plecanatide belongs to a class termed as Uroguanylin analog. These agents increase the activation of guanyl cyclase C in the luminal area of intestines. Increase activation of guanyl cyclase C gives rise to fluid retention, intestinal motility and rapid intestinal transit time.

Constipation is very common problem in among all kind of populations. Constipation may occur in any person without regard to age or gender. There are number of reasons that trigger constipation problem like low or no fluid intake, impaired intestinal absorption, impaired intestinal motility, chronic disease of intestine, lack of fiber in the diet and last but not the least side effect of drugs like morphine.

Basic mechanism by which Trulance/plecanatide tones up intestinal motility is that it increases the activation of guanyl cyclase C (GC-C) in the luminal area of intestines. Such rise in activation of GCC leads to increase in cyclic guanosine monophosphate (cGMP), anion secretion,  fluid secretion, and intestinal transit. In this way it subsides the symptoms of constipation.


Trulance/Plecanatide Cost, Side effects, Dosage, Uses for Chronic Idiopathic Constipation (CIC)


Trulance/Plecanatide Side effects

According to clinical data provided by health authorities this drug is not suitable for children having age below six years. This drug withdraws some amount of water from intestines thus it will increase the risk of dehydration especially in children. In adult, following side effects were observed during clincal trails.
  • Dehydration
  • Severe Diarrhea
  • Intestinal Discomfort
  • Colic Pain
  • Increase in bowel motility
  • Headache
  • Nausea

Trulance/Plecanatide Uses or Prescribing Information

U.S FDA approved this drug for the treatment of chronic constipation of unknown origin (idiopathic) in adults. If patient is suffering from constipation due to known cause like low or no fluid intake, impaired intestinal motility or drug induced constipation then this drug should be avoided and root cause treatment should be followed. This drug is taken orally.

Trulance/Plecanatide Dosage


Standar dosage frequency of trulance in adults is 3mg once daily. However, its safety and efficacy has not yet evaluated in patients having age 6 yrs to <18 yrs

Trulance/Plecanatide Cost and Dosage


According to market survey this drug is currently not available at retail pharmacies because it is recently approved by FDA and is under manufacturing process. Once, it will be available at retail outlets we will let you know about the unit cost of drug and its dosage frequency.

Spinraza/nusinersen Cost, Side effects, Dosage, Uses for spinal muscular atrophy

Spinraza is marketed by Biogen of Cambridge, Massachusetts and was developed by Ionis Pharmaceuticals of Carlsbad, California.

U.S FDA approved new medicine spinraza that contains active ingredient nusinersen  for the treatment of rare muscular disorder known as spinal muscular atrophy (SMA). FDA approves this drug in parenteral dosage form and a medical specialist is required to inject this drug because it is administered  into the fluid surrounding the spinal cord.

Spinal muscular atrophy is a rare kind of genetic disorder. Motor neurons are important to control motor functions of body like moving, driving, various muscular movements etc. Increase in number of dead motor neurons makes the co-ordination weak between brain and skeletal muscles. This will ultimately leads to the development of SMA (spinal muscular atrophy). However, certain risk factors that play important role in the development of SMA (spinal muscular atrophy) are genetic predisposition, age factor, auto-immune disease etc.

This drug belongs to antisense oligonucleotide family and can be used in all types of spinal muscular atrophy. It is injected through intrathecal route so, much care is required during its administration in order to avoid nerve damage. This drug has been received orphan drug designation which provides incentives to assist and encourage the development of drugs for rare diseases.


Spinraza/nusinersen Cost, Side effects, Dosage, Uses for spinal muscular atrophy

Spinraza/nusinersen Side effects

During initial clinical trials drug shows following side effects
  • Thromobocytopenia (low platelets count) 
  • Anemia (low RBC's count)
  • Increased risk of respiratory tract infection
  • Kidney damage (nephrotoxicity)
  • Brain damage (neurotoxicity)
  • Constipation
  • Headache
  • Dizziness
  • Hypersensitivity reactions
  • Skin rashes
  • Painful administration

Spinraza/nusinersen Uses or Prescribing information

U.S FDA approved this drug for all kinds of spinal muscular atrophy (SMA). This drug should be used once every 4 months after loading dose administration via intrathecal route. Care should be taken while injecting this drug to avoid any nerve damage.

Spinraza/nusinersen Dosage

This drug is manufacturing as single dose vial with potency of 12mg/5mL. Dosage of spinraza is diving into two phases.

Loading Dose: It includes 4 doses each of 12mg intrathecally. Administer first 3 doses at 14-day intervals and then administer 4th dose 30 days after the last (3rd) dose.

Maintainance Dose: It includes dose of 12 mg intrathecally per administration after every 4 months.

Spinraza/nusinersen Cost

This drug is administered by your healthcare practitioner (HCP), which usually means:
  • It may comes under special items category.
  • You cannot fill this prescription in a regular pharmacy. Your doctor, hospital, or clinic will provide this  medication because of possible complications or other issues.
  • This drug is likely to be covered under your medical benefit if you have insurance.

Rubraca/rucaparib Cost, Side effects, Dosage, Uses for advanced ovarian cancer

Rubraca is marketed by Clovis Oncology, Inc. based in Boulder, Colorado. 

U.S FDA assigned accelerated approval pathway to new medicine Rubraca that contains active ingredient rucaparib for the treatment of various types of ovarian cancer. According to FDA guidelines this drug should be used in progressive or advance type of ovarian cancer.

According to statistical data ovarian cancer is one of most leading type of cancer in women with high mortality rate. Ovarian cancer has multiple types some of which are idiopathic while others are due to mutation in specific genes e.g BRCA gene. Statistics shows that in United states approximately 20,000 to 22,000 women diagnosed with ovarian cancer annually and about 60 to 70 percent of these women die due to ovarian cancer. In united states most majority of women have ovarian cancer due to mutation of BRCA gene.

Rubraca/rucaparib achieved accelerated approval pathway due to its targeted action. Normally BRCA genes involves in the development of DNA but due to mutation in BRCA genes they produce altered DNA and give rise to tumor cells. rubraca/rucaparib works by blocking the enzyme (polymerase) that is necessary for DNA replication. This blockade will restrict the process of DNA replication or repairing, leading to cell death and possibly a slow-down or stoppage of tumor growth.

Rubraca/rucaparib Cost, Side effects, Dosage, Uses for advanced ovarian cancer


Rubraca/rucaparib Side effects


During clinical trails following side effects were reported in patient who were receiving this medicine

  • Low platelets count
  • Decrease in red blood cells
  • Difficulty in breathing
  • Changes in taste sensation
  • Fatigue
  • Nausea
  • Vomiting
  • Skin rashes

Rubraca/rucaparib Uses or Prescribing Information


U.S FDA granted its use in women who have advance ovarian cancer and one or more of the mutations of BRCA genes are detected, the patient may be eligible for treatment with Rubraca. However drug should be used with caution in patients who have bone marrow problem because this drug may cause myelodysplastic syndrome in those patients. This drug is not safe for fetus development so, always consult with your health care provide (hcp) before starting any medicine.

Rubraca/rucaparib Dosage


This drug is manufacturing in the form of tablets (Oral dosage form) with potency of 200mg and 300mg. Standard dosage frequency of Rubraca/rucaparib is 300mg tablet twice a day (BID) daily until disease progression reduced to maximum level. Dose adjustment may be required in case of severe side effects, hepatic or renal impairment. Safety profile of this drug has not yet evaluated in children.

Rubraca/rucaparib Cost 

This drug is now available in the market with an average cost of 3900$ to 3700$ per pack. There two potencies of rubraca 200mg and 300mg available in the market. Both have same pack size (30's). This price may variate with the passage of time so, make it confirm by visiting your nearby pharmacy before purchasing.

Eucrisa/crisaborole Cost, Side effects, Dosage, Uses for Eczema

Eucrisa is manufactured by Palo Alto, California-based Anacor Pharmaceuticals, Inc.

U.S FDA approved new drug Eucrisa that contains crisaborole as topical dosage form  (ointment) for the treatment of eczema ( atopic dermatitis) in patients having age 2 years or more. Eczema. Eczema or atopic dermatitis is a common skin disorder among all populations. It is intense form of skin disorder with an inflammatory response that requires long-term management or sometimes life time management in advance stages.

Exact underlying cause of eczema is not fully understood that triggers inflammatory response. It is proposed that genetic predisposition, environmental factors and immunogenic response play key role in the parthenogenesis of eczema. Most common symptoms of eczema include dry and red scaly appearance of affected area of skin with intense itching. Scratching may cause skin thickness along with swelling that prolongs the duration of therapy.

Main mechanism of Eucrisa/crisaborole is inhibition of  phosphodiesterase-4 that causes inflammation by triggering the release of certain inflammatory mediators like interleukin-12(IL-12), bradykinins, cytokinins and tumor necrosis factor (TNFα). As name indicates that it contains boron that penetrates readily and rapidly into skin is essential for its binding activity.

Eucrisa/crisaborole Cost, Side effects, Dosage, Uses for Eczema

Eucrisa/crisaborole Side effects

According to clinical data provided by health authorities following side effects were documented during clinical trials

  • Hypersensitivity reactions
  • Burning sensation
  • Sting like feeling
  • Mild pain during application

Eucrisa/crisaborole Uses or Prescribing information

U.S FDA approved only topical form of Eucrisa/crisaborole for the treatment eczema ( atopic dermatitis). Clinical data suggests that it should be applied two times a day to get better results. Eucrisa/crisaborole should not be used in patients having age less than 2 years.

Eucrisa/crisaborole Dosage

Eucrisa/crisaborole is manufacturing in the form of ointment with potency of 2%. Normally it should be applied in the form of thin layer topically to affected area twice a day. It should be used in case of mild to moderate kind of atopic dermatitis. It should not be used in children having age less than 2 years.

Eucrisa/crisaborole Cost

Eucrisa is now avaiable in the market in two pack size 60g and 100g. Average cost of 1 tube (60g) of eucrisa with 2% potency ranges between 500$ - 800$. This price may variate with the passage of time so, make it confirm by visiting your nearby pharmacy before purchasing.

Intrarosa/prasterone Cost, Side effects, Dosage, Uses for intercourse Pain in Women

Intrarosa is marketed by Quebec-based Endoceutics Inc.

U.S. FDA approved a new product Intrarosa that contains active ingredient prasterone for the treatment of pain in postmenopausal women during sexual intercourse. This pain intensity might be moderate to severe and condition is known as dyspareunia. Intrarosa is first kind of this category approved by FDA. Prosterone is basically a steroidal hormone that produces naturally inside body ( endogenous Production). It is also know as Dehydroepiandrosterone (DHEA) and circulates in human body in large amounts. Prosterone plays many physiological role in the body like biosynthesis of sex hormones like estrogens and androgens.

Postmenopuasal problems are very common in women like flushing, mood swings, metabolic changes in the body and osteoporosis. According to research major underlying cause of intense pain during sexual intercourse in postmenopausal women is atrophy of internal membrane of vulva and vagina. It is proposed that this atrophy is triggered due to low levels of estrogen circulating in the body and vaginal tissues for a long time and condition is known as vulvar and vaginal atrophy (VVA).

Basic mechanism of Intrarosa (prasterone) is that it decreases the adrenal insufficiency in women but still exact pathway by which it works is not fully understood due to inadequate clinical data. According to FDA its efficacy was established after 12 week clinical trials and women who were taking intrarosa during these trials experienced remarkable decrease in the severity of pain.


Intrarosa/prasterone Side effects 

According to data obtained from clinical trials all side effects were minor to moderate. During these clinical trials no life threatening or lethal side effect was reported. Detail of these side effects is as follows

Minor Side effects

  • Headache
  • Irritation during insertion
  • Sense of discomfort in lower abdominal

Moderate Side effects

  • Vaginal Discharge
  • Abnormal Pap smear
  • Vaginal pain
  • Vaginal odor

Intrarosa/prasterone Uses or Prescribing Information

Though, prasterone is available in many dietary supplements but FDA approved only vaginal pessaries of prasterone to decrease the intensity of pain during intercourse in postmenopausal women. According to clinical trials intrarosa shows its therapeutic effect when used for specified period of time. Short term usage for few days does not produce significant effect.

Intrarosa/prasterone Dosage

Intrarosa is manufacturing in the form of vaginal insert tab or pessary. Each tab or pessary contains 6.5mg of prasterone for the treatment of moderate to severe dyspareunia caused by postmenopausal vulvar/vaginal atrophy. Standard dosage frequency of intrarosa in women is 1 vaginal insert daily at bedtime with the help of applicator.

Intrarosa/prasterone Cost

According to market survey this drug is currently not available at retail pharmacies because it is recently approved by FDA and is under manufacturing process. Once, it will be available at retail outlets we will let you know about the unit cost of drug and its dosage frequency.
 

Lartruvo/olaratumab Cost, Side effects, Dosage, Uses for soft tissue sarcoma (STS)

Lartruvo is marketed by Eli Lilly and Company based in Indianapolis, Indiana.


FDA has approved a new drug Lartruvo for soft tissue sarcoma. This drugs contains active ingredient olaratumab which monoclonal antibody and extracted from human origin.Monoclonal antibodies are derived from cells of immune system and have high affinity for binding to specific site. Monoclonal antibodies are commonly usedin chemotherapy to target specific site and minimize the chances of toxicity.

FDA recommended the usage of newly approved drug Lartruvo (olaratumab) in combination with doxorubicin to make soft tissue sarcoma treatment more effective.According to guidelines provided by FDA this drug along with doxorubicin should only be used in those patients who are not recovering after radiation or surgery.

Soft tissue sarcoma (STC) is a kind of tumor that originates from connective tissues. There are number of underlying factors that contribute towards development of soft tissue sarcoma. For example excessive exposure to toxic substances in chemical industries, severe viral infection, genetic predisposition, environmental exposure or pollution etc.

Basic mechanism of Lartruvo (olaratumab) is that it blocks platelet derived growth factor (PDGF) receptor alpha that increases the growth of soft tissue sarcoma. PDGF is basically a protein that contributes in tumor growth. Latruvo decreases the progression of this disease by blocking these proteins.


Lartruvo/olaratumab Cost, Side effects, Dosage, Uses for soft tissue sarcoma (STS)


Lartruvo/olaratumab Side effects

According to data received from clinical trails following side effects were observed initially,

  • Neuropathy or Nerve damage
  • Baldness
  • Loss of appetite
  • Low white blood cells count (Neutropenia)
  • Fetal abnormalities
  • Reactions during infusion
  • Blood pressure fluctuations
  • Asthenia
  • Swelling of mucous membranes
  • Headache
  • Musculoskeletal pain
  • Nausea
  • Vomiting
  • Abdominal discomfort

Lartruvo/olaratumab prescribing information/uses

According to data provided by U.S FDA this drug is reserved for those patients who are not responding to radiations or surgery. FDA also recommended that this drug should be used in combination with doxorubicin to make the treatment more targeted and effective.

Lartruvo/olaratumab Dosage

This drug is formulated as a 50ml vial for intravenous use with potency of 10mg/ml. Standard dosage of lartruvo for the treatment of soft tissue sarcoma is 15mg/Kg intravenously at rate of 1-8hrs.Dose adjustment can be done depending upon the patient condition and disease progression.

Lartruvo/olaratumab Cost

This drug is administered by your healthcare practitioner (HCP), which usually means:
  • It may comes under special items category.
  • You cannot fill this prescription in a regular pharmacy. Your doctor, hospital, or clinic will provide this  medication because of possible complications or other issues.
  • This drug is likely to be covered under your medical benefit if you have insurance.

Amjevita/adalimumab-atto Cost, Side effects, Dosage, Uses for multiple inflammatory diseases

Amjevita is manufactured by Amgen, Inc., of Thousand Oaks, California.

The U.S. Food and Drug Administration has approved a new biosimilar drug Amjevita that contains adalimumab-atto as an active pharmaceutical ingredient.Amjevita is a biosimilar to previously approved drug Humira manufactured by AbbVie Inc. of North Chicago, Illinois.

   
Amjevita/adalimumab-atto basic mechanism is inhibition or suppression tumor necrosis factor alpha (TNFa) which is main culprit for inflammatory response in inflammatory diseases (auto-immune) like rheumatoid arthritis, psoriatric arthritis, ankylosing spondylitis etc.It was observed during clinical trial that administration of adalimumab and methotrexate (MTX) provide synergistic effect and doubles the therapeutic efficacy of methotrexate. Apart from these indications Amjevita/adalimumab-atto can also be used in a patho-physiological condition where suppression of immune system is required. This drug is administered through subcutaneous route (sc).

Multiple inflammatory diseases is a group of disorders that belongs to malfunctioning of immune system. Most common examples of these diseases are rheumatoid arthritis, ulcerative colitis, crohn's disease, hidradenitis suppurativa, juvenile idiopathic arthritis. All kinds of auto-immune disorders are treated and managed by following combination of therapy like DMARD's, Immunosuppressants, Steroidal drugs, etc.

Amjevita/adalimumab-atto Cost, Side effects, Dosage, Uses for multiple inflammatory diseases

 Amjevita/adalimumab-atto Side effects


Amjevita is a biosimilar of Humira  so, both have almost same kind of indications and side effects. These side effects can be categorized into moderate to severe.

Moderate Side effects 

  • Generalized weakness
  • feeling of numbness
  • Body pain
  • Shortness of breath
  • vision problems
  • skin rashes
  • swelling on body

Severe Side effects

Severe kind of side effects associated with this drugs are increase risk of

  • Infections like respiratory infection, liver and kidney infection.
  • Malignancy  

Amjevita/adalimumab-atto Uses

U.S FDA approved this drug for multiple inflammatory diseases which is a class of auto-immune disorders. Auto-immune disorders are of many types like rheumatoid arthritis, ulcerative colitis, crohn's disease, hidradenitis suppurativa, juvenile idiopathic arthritis. This drug can also be used in combination with other drugs like methotrexate.

Amjevita/adalimumab-atto Dosage

This drug is formulated as pre-filled syringe with potency of 40mg/0.8ml. Standard dosage of amevita/adalimumab  is 40mg after every two weeks. It is administered subcutaneously and dose may be adjusted depending upon the severity of disease and patient condition. During this therapy your health care provider may also prescribe you some other DMARD's like methotrexate to make the treatment more effective.

Amjevita/adalimumab-atto Cost 

According to market survey this drug is currently not available at retail pharmacies because it is recently approved by FDA and is under manufacturing process. Once, it will be available at retail outlets we will let you know about the unit cost of drug and its dosage frequency.

Exondys 51/eteplirsen Cost, side effects, Mechanism, Dosage for Duchenne muscular dystrophy (DMD)

Exondys 51 is made by Sarepta Therapeutics of Cambridge, Massachusetts.

US FDA approved a new novel drug Exondys 51 for the treatment of Duchenne muscular dystrophy (DMD). This drug contains eteplirsen as an active ingredient. Exondys 51/eteplirsen specifically targets mutations that occurs in DMD gene.This drug has been approved only for the treatment and management of Duchenne muscular dystrophy (DMD) but it does not mean that it cures it forever.

Duchenne muscular dystrophy (DMD) is rare kind of disorder but it is very devastating degenerative disease. In this disease some mutational changes occur in DMD gene which leads to alteration in DMD RNA transcription process. This altered DMD RNA becomes non-functional or in-active and does not bind or recognize dystrophin protein. This is the main root cause of Duchenne muscular dystrophy (DMD).

Exondys 51/eteplirsen Mechanism of Action or Pharmacology


Exondys 51/eteplirsen is  morpholino antisense oligomer that causes cleavage of exon 51 (part of a gene that will encode a part of the final mature RNA) during pre mRNA splicing phase of dystrophin protein.This process will generate a functional but modified form of dystrophin protein (skipping exon therapeutically) that can reinstate the binding process that was impaired due to genetic mutations.

The whole process initiated by this drug does not cure this problem but reduce the severity, progression and degeneration. Majority of patients suffering from DMD have this kind of mutation and can be treated effectively with the help of this therapeutic drug Exondys 51/eteplirsen.

Exondys 51/eteplirsen Cost, side effects, Mechanism, Dosage for Duchenne muscular dystrophy (DMD)


Exondys 51/eteplirsen Side effects

According to clinical trials this drug exhibited some minor side effects but no life threatening side effect was reported at that time. These minor side effects were

  • Balance disorder
  • Nausea
  • Vomiting
  • Headache
  • Dizziness
  • palpitation

This drug is administered in form of infusion through intra-venous route only.

Exondys 51/eteplirsen Dosage

This drug is formulated as a single dose vial with potency of 50mg/ml. In adult DMD patients  its standard dosage is 30mg/Kg through intravenous route once in a week. Dose titration may be considered depending upon the patient condition and severity of disease. 

Exondys 51/eteplirsen Cost

This drug is administered by your healthcare practitioner (HCP), which usually means:
  • It may comes under special items category.
  • You cannot fill this prescription in a regular pharmacy. Your doctor, hospital, or clinic will provide this  medication because of possible complications or other issues.
  • This drug is likely to be covered under your medical benefit if you have insurance.