Orkambi Side Effects

 There may be nausea, gas or diarrhea. If these effects persist or worsen, please inform your doctor or pharmacist immediately.

Remember, your doctor prescribes this medicine because he or she believes that the benefits to you outweigh the risks of side effects. Many people who use this drug do not have serious side effects.

This medication may increase blood pressure. Check your blood pressure regularly and tell your doctor if the result is high.

If you have any serious side effects, please tell your doctor immediately, including: vision changes.

This drug may rarely cause serious liver disease. If you have any symptoms of liver damage, seek medical help immediately, including: nausea/vomiting, loss of appetite, stomach/abdominal pain, yellow eyes/skin, dark urine.

Some people who already have severe lung disease may have breathing problems after starting this medication. If so, seek medical help immediately: chest pain, shortness of breath.

Lumacaftor/ivacaftor usually causes a rash, which is usually not serious. However, you may not be able to tell it apart from the rare rash, which may be a sign of a serious reaction. If a rash occurs, tell the doctor immediately.

Very severe allergic reactions to this drug are rare. However, if you notice any symptoms of a severe allergic reaction, including rash, itching/swelling (especially face/tongue/throat), severe dizziness, and difficulty breathing, please seek medical help immediately.

This is not a complete list of possible side effects. If you notice other effects not listed above, please contact your doctor or pharmacist.

Orkambi Uses

 The drug is a combination of 2 drugs: lumacaftor and ivacaftor. It is used to treat cystic fibrosis in some people (people with 2 abnormal copies of the "CFTR" gene). It may help improve breathing, reduce the risk of lung infections and improve weight.
How to use Orkambi

Please read the "Patient Information Booklet" provided by the pharmacist before starting to take lumacaftor / ivacaftor and every time you supplement. If you have any questions, please consult your doctor or pharmacist.

Follow your doctor's instructions to take this medication by mouth, usually twice a day (12 hours apart). Eat fat-containing meals or snacks before or immediately after taking the medicine. Examples include eggs, avocados, nuts, butter, peanut butter, cheese pizza or whole milk products (such as whole milk, cheese, yogurt).

If you are taking this medicine in granular form, shake the package gently before use. Open the package and carefully pour the contents into a small bowl containing 1 teaspoon (5ml) of soft food or liquid (such as yogurt, applesauce, pudding, milk or juice). Soft food or liquids should be at or below room temperature. After mixing, absorb all the mixture within 1 hour. Make sure to eat fatty foods as directed.

The dosage depends on your age, medical condition, response to treatment, and other medications you may be taking. Be sure to inform your doctor and pharmacist of all products you use (including prescription drugs, over-the-counter drugs, and herbal products).

Use this medication regularly to get the most benefit. To help you remember, take it at the same time every day. If you temporarily stop taking this medicine for more than 1 week, please consult your doctor for specific instructions on restarting this medicine. If you are taking certain other medications, you may be instructed to restart at a lower dose in the first week.

DEFITELIO DOSAGE AND ADMINISTRATION

 The recommended dosage of DEFITELIO for adult and pediatric patients is 6.25 mg/kg every 6 hours given as a 2- hour intravenous infusion. The dose should be based on patient's baseline body weight, defined as the patient's weight prior to the preparative regimen for HSCT.

Administer DEFITELIO for a minimum of 21 days. If after 21 days signs and symptoms of hepatic VOD have not resolved, continue DEFITELIO until resolution of VOD or up to a maximum of 60 days.
Administration Instructions

    DEFITELIO must be diluted prior to infusion [see Preparation Instructions].
    Prior to administration of DEFITELIO, confirm that the patient is not experiencing clinically significant bleeding and is hemodynamically stable on no more than one vasopressor [see WARNINGS AND PRECAUTIONS].

    Administer DEFITELIO by constant intravenous infusion over a 2-hour period.
    Administer the diluted DEFITELIO solution using an infusion set equipped with a 0.2 micron in-line filter. Flush the intravenous administration line (peripheral or central) with 5% Dextrose Injection, USP or 0.9% Sodium Chloride Injection, USP immediately before and after administration.
    Do not co-administer DEFITELIO and other intravenous drugs concurrently within the same intravenous line.

Preparation Instructions

Dilute DEFITELIO in 5% Dextrose Injection, USP or 0.9% Sodium Chloride Injection, USP to a concentration of 4 mg/mL to 20 mg/mL. Administer the diluted solution over 2 hours.

Vials contain no antimicrobial preservatives and are intended for a single-patient-use only. Partially used vials should be discarded. Use the diluted DEFITELIO solution within 4 hours if stored at room temperature or within 24 hours if stored under refrigeration. Up to four doses of DEFITELIO solution may be prepared at one time, if refrigerated.
Preparation Instructions

    Determine the dose (mg) and number of vials of DEFITELIO based on the individual patient's baseline weight (weight prior to the preparative regimen for HSCT).
    Calculate the volume of DEFITELIO needed, withdraw this amount from the vial(s) and add it to the infusion bag containing 0.9% Sodium Chloride Injection or 5% Dextrose Injection for each dose to make a final concentration of 4 mg/mL to 20 mg/mL.
    Gently mix the solution for infusion.
    Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Only clear solutions without visible particles should be used. Depending on the type and amount of diluent, the color of the diluted solution may vary from colorless to light yellow.

DEFITELIO

 Defibrotide sodium is an oligonucleotide mixture with profibrinolytic properties. The chemical name of defibrotide sodium is polydeoxyribonucleotide, sodium salt. Defibrotide sodium is a polydisperse mixture of predominantly single-stranded (ss) polydeoxyribonucleotide sodium salts derived from porcine intestinal tissue having a mean weighted molecular weight of 13-20 kDa, and a potency of 27-39 and 28-38 biological units per mg as determined by two separate assays measuring the release of a product formed by contact between defibrotide sodium, plasmin and a plasmin substrate. The primary structure of defibrotide sodium is shown below.

DEFITELIO (defibrotide sodium) injection is a clear, light yellow to brown, sterile, preservative-free solution in a single-patient-use vial for intravenous use. Each milliliter of the injection contains 80 mg of defibrotide sodium and 10 mg of Sodium Citrate, USP, in Water for Injection, USP. Hydrochloric Acid, NF, and/or Sodium Hydroxide, NF, may have been used to adjust pH to 6.8-7.8.

INDICATIONS

DEFITELIO is indicated for the treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT).

Main Types of ASL

 
What Are the Main Types?

There are two types of ALS:

Sporadic ALS is the most common form. It affects up to 95% of people with the disease. Sporadic means it happens sometimes without a clear cause.

    Familial ALS (FALS) runs in families. About 5% to 10% of people with ALS have this type. FALS is caused by changes to a gene. Parents pass the faulty gene to their children. If one parent has the gene for ALS, each of their children will have a 50% chance of getting the gene and having the disease.

What Causes ALS?

Researchers still don't know exactly what causes motor neurons to die with ALS. Gene changes, or mutations, are behind 5% to 10% of ALS cases. More than 12 different gene changes have been linked to ALS.

One change is to a gene that makes a protein called SOD1. This protein may be toxic to motor neurons. Other gene changes in ALS might also damage motor neurons.

Environment could also play a role in ALS. Scientists are studying whether people who come into contact with certain chemicals or germs are more likely to get the disease. For example, people who served in the military during the 1991 Gulf War have gotten ALS at higher rates than usual.

What Is ALS? What Are the Types and Causes?

 

ALS is short for amyotrophic lateral sclerosis. You might also have heard it called Lou Gehrig's disease, after the baseball player who was diagnosed with it in the 1930s. A French doctor named Jean-Martin Charcot discovered the condition in 1869.

ALS is a progressive disease, which means it gets worse over time. It affects nerves in your brain and spinal cord that control your muscles. As your muscles get weaker, it gets harder for you to walk, talk, eat, and breathe.
ALS and Motor Neurons

It’s a disease that affects your motor neurons. These nerve cells send messages from your brain to your spinal cord and then to your muscles. You have two main types:

    Upper motor neurons: Nerve cells in the brain.
    Lower motor neurons: Nerve cells in the spinal cord to muscle.

These motor neurons control all your voluntary movements -- the muscles in your arms, legs, and face. They tell your muscles to contract so you can walk, run, pick up your smartphone, chew and swallow food, and even breathe.

ALS is one of a few motor neuron diseases. Some others include:

    primary lateral sclerosis (PLS)
    progressive bulbar palsy (PBP)
    pseudobulbar palsy

What Happens When You Have ALS

With ALS, motor neurons in your brain and spinal cord break down and die.

When this happens, your brain can't send messages to your muscles anymore. Because the muscles don't get any signals, they become very weak. This is called atrophy. In time, the muscles no longer work and you lose control over their movement.

At first, your muscles get weak or stiff. You may have more trouble with fine movements -- such as trying to button a shirt or turn a key. You may stumble or fall more than usual. After a while, you can't move your arms, legs, head, or body.

Eventually, people with ALS lose control of their diaphragm, the muscles in the chest that help you breathe. Then they can't breathe on their own and will need to be on a breathing machine.

The loss of breathing causes many people with ALS to die within 3 to 5 years after they're diagnosed. Yet some people can live more than 10 years with the disease.

People with ALS can still think and learn. They have all of their senses -- sight, smell, hearing, taste, and touch. Yet the disease can affect their memory and decision-making ability.

ALS isn't curable. Yet scientists now know more about this disease than ever before. They are studying treatments in clinical trials.

ORENCIA DOSAGE AND ADMINISTRATION

 Adult Rheumatoid Arthritis

For adult patients with RA, ORENCIA may be administered as an intravenous infusion or as a subcutaneous injection.

ORENCIA may be used as monotherapy or concomitantly with DMARDs other than TNF antagonists.
Intravenous Dosing Regimen

ORENCIA lyophilized powder should be reconstituted and administered after dilution [see Adult Psoriatic Arthritis] as a 30-minute intravenous infusion utilizing the weight range-based dosing specified in Table 1. Following the initial intravenous administration, an intravenous infusion should be given at 2 and 4 weeks after the first infusion and every 4 weeks thereafter.

ORENCIA 125 mg in prefilled syringes or in ORENCIA ClickJect™ autoinjector should be administered by subcutaneous injection once weekly [see Preparation And Administration Instructions For Intravenous Infusion] and may be initiated with or without an intravenous loading dose. For patients initiating therapy with an intravenous loading dose, ORENCIA should be initiated with a single intravenous infusion (as per body weight categories listed in Table 1), followed by the first 125 mg subcutaneous injection administered within a day of the intravenous infusion.

Patients transitioning from ORENCIA intravenous therapy to subcutaneous administration should administer the first subcutaneous dose instead of the next scheduled intravenous dose.

Juvenile Idiopathic Arthritis

For patients with juvenile idiopathic arthritis (JIA), ORENCIA may be administered as an intravenous infusion (6 years of age and older) or a subcutaneous injection (2 years of age and older). Intravenous dosing has not been studied in patients younger than 6 years of age.

ORENCIA may be used as monotherapy or concomitantly with methotrexate.
Intravenous Dosing Regimen

ORENCIA should be administered as a 30-minute intravenous infusion based on body weight. Pediatric patients with:

    body weight less than 75 kg should be administered ORENCIA at a dose of 10 mg/kg [see Adult Psoriatic Arthritis].
    body weight of 75 kg or more should be administered ORENCIA following the adult intravenous dosing regimen (see Table 1), not to exceed a maximum dose of 1000 mg.

Following the initial administration, ORENCIA should be given at 2 and 4 weeks after the first infusion and every 4 weeks thereafter. Any unused portions in the vials must be immediately discarded.

ORENCIA

ORENCIA® (abatacept) is a selective T cell costimulatory modulator. ORENCIA is a soluble fusion protein consisting of the extracellular domain of human cytotoxic T lymphocyte-associated antigen 4 (CTLA-4), which is combined with the modified Fc (hinge, CH2 and CH3 domains) of human immunoglobulin G1 (IgG1) Partially connected. Abatacept is produced in a mammalian cell expression system through recombinant DNA technology. The apparent molecular weight of abatacept is 92 kilodaltons.

ORENCIA injection is a lyophilized powder for intravenous infusion. ORENCIA injection is a sterile, white, preservative-free lyophilized powder that can be reconstituted and diluted before intravenous administration. After rebuilding the lyophilized powder with 10 mL of sterile water for injection (USP), the OERNCIA solution was clear, colorless to light yellow, and the pH was 7.2 to 7.8. Each single-use ORENCIA injection bottle provides 250 mg of abatacept, maltose (500 mg), sodium dihydrogen phosphate (17.2 mg) and sodium chloride (14.6 mg) for administration.

ORENCIA injection is a sterile, preservative-free, transparent to slightly opalescent, colorless to light yellow solution, and the pH range for subcutaneous administration is 6.8 to 7.4. ORENCIA injection is provided in the form of a single-dose prefilled syringe or a single-dose ClickJect auto-injector.

ORENCIA® is suitable for reducing the symptoms and signs of adult patients with moderate to severe active rheumatoid arthritis, causing major clinical reactions, inhibiting the progression of structural damage and improving physical function. In addition to tumor necrosis factor (TNF) antagonists, OERNCIA can also be used as a monotherapy or in conjunction with disease-modifying anti-rheumatic drugs (DMARD).
Juvenile idiopathic arthritis

ORENCIA is indicated for alleviating signs and symptoms of adolescent idiopathic arthritis with moderately to severely active polyarticular joints aged 2 years and older. ORENCIA can be used as a monotherapy or in combination with methotrexate (MTX).
Adult Psoriatic Arthritis (PsA)

ORENCIA is designated for the treatment of adult patients with active psoriatic arthritis (PsA).
Important usage restrictions

ORENCIA should not be used simultaneously with TNF antagonists. It is not recommended to use ORENCIA with other biological rheumatoid arthritis (RA) treatments (such as anakinra).