How Do Other Metabolic and Endocrine Drugs Work?

Metabolic and endocrine drugs are used to treat disorders associated with the endocrine system and metabolism. The endocrine system is a network of glands that secrete hormones, responsible for the normal functioning of every body organ. Metabolism refers to the process by which the body converts food into energy and removes wastes from the body.

Medications that do not fall into any specific class of metabolic and endocrine drugs are categorized as other metabolic and endocrine drugs. Other metabolic and endocrine drugs include the following:

• Becaplermin: Becaplermin is a gel applied topically to treat skin ulcers caused by diabetic neuropathy (nerve damage). Beclapermin is produced by recombinant DNA technology, and the medication works like a human platelet-derived growth factor (PDGF). Becaplermin promotes the recruitment and proliferation of cells involved in wound repair and enhances new tissue formation, helping the ulcer heal.
• Betaine: Betaine is used to treat homocystinuria, an inherited disorder of methionine metabolism, which leads to excessive buildup of a compound known as homocysteine in blood and urine. Methionine is an essential amino acid required for protein synthesis. Betaine helps convert homocysteine into methionine.
• Bromocriptine: Bromocriptine is a dopamine receptor agonist used to treat disorders associated with dopamine dysfunction. Dopamine is an important hormone/neurotransmitter that has a multitude of functions including regulation of movement, and activity of hormones such as prolactin and growth hormones. Bromocriptine stimulates dopamine receptors in the brain and enhances dopamine activity.
• Carnitine (levocarnitine): Carnitine is a natural compound in the body that is essential for transporting free fatty acids into the cellular mitochondria and converting them to energy. Carnitine is prescribed as a supplement to people who have carnitine deficiency, and to end-stage renal disease patients who usually have abnormal carnitine metabolism.
• Cerliponase alfa: Cerliponase alfa is a purified form of the human enzyme tripeptidyl peptidase-1, which breaks up peptides into amino acids. Cerliponase alfa is used to supplement the natural deficiency in tripeptidyl peptidase-1 in infantile neuronal ceroid lipofuscinosis type 2, an inherited disorder that causes abnormal storage of proteins and fats, and primarily affects the nervous system.
• Cysteamine: Cysteamine is used to treat nephropathic cystinosis, a metabolic disorder in which cystine, an amino acid accumulates in the lysosomes, digestive units within cells, crystallizes, and eventually damages the cells. Cysteamine converts lysosomal cystine into cysteine and to cysteine-cysteamine mixed disulfide which can exit the cell.
• Dextrose (glucose): Dextrose is sugar from corn or wheat, almost identical to glucose, the sugar found in the bloodstream. Dextrose is administered intravenously to treat low blood glucose levels (hypoglycemia).
• Dichlorphenamide: Dichlorphenamide is a medication used to treat periodic paralysis and muscle weakness caused by high or low levels of potassium in the blood (hyperkalemia/hypokalemia). Dichlorphenamide belongs to the class carbonic anhydrase inhibitors drug class, and it helps normalize potassium levels, however, its exact mechanism of action in periodic paralysis is unknown.
• Eliglustat: Eliglustat is a medication used to treat Gaucher disease, a condition caused by deficiency of glucocerebrosidase, a lysosomal enzyme, which causes accumulation of glucocerebroside, a type of fat, in the cells. Eliglustat inhibits the enzyme glucosylceramide synthase and blocks the synthesis of glucocerebroside.
• Nitisinone: Nitisinone is a medication used to treat hereditary tyrosinemia type 1, a disorder caused by a genetic mutation in the fumarylacetoacetate hydrolase (FAH) gene, which results in deficiency of the FAH enzyme.
  • FAH enzyme is one of the enzymes required for the proper breakdown of tyrosine, an essential amino acid in most proteins. FAH enzyme deficiency causes tyrosine to break down into toxic metabolites. Nitisinone blocks the breakdown of tyrosine upstream of the FAH enzyme and prevents the accumulation of toxic metabolites.
• Pegvisomant: Pegvisomant is a medication used to treat acromegaly, a disorder in which adults have excessive bone growth in the face, hands, and feet due to high levels of growth hormone. Pegvisomant is a lab-manufactured protein, using DNA recombinant technology. Pegvisomant binds to growth factor receptors on cell surfaces and blocks natural growth hormone from stimulating growth.
• Triheptanoin: Triheptanoin is a medium-chain triglyceride used in the treatment of long-chain fatty acid oxidation disorders (LC-FAOD). LC-FAOD is genetic metabolic disorders with enzyme deficiencies that prevent the breakdown of fatty acids into energy. Triheptanoin bypasses the LC-FAOD enzymes to provide an alternate source of calories and fatty acids, to people with LC-FAOD.
• Vestronidase alfa-vjbk: Vestronidase alfa-vjbk is a purified human enzyme beta-glucuronidase (GUS) used to treat mucopolysaccharidosis VII, a lysosomal disorder with GUS deficiency that causes accumulation of glycosaminoglycans (GAG), leading to multisystem tissue and organ damage. Vestronidase replaces the natural GUS enzyme deficiency and helps break down the accumulated glycosaminoglycans.

Other metabolic and endocrine drugs may be administered through different routes including:

• Oral: Tablets, capsules, granules, solutions, liquids, and suspensions
• Topical: Gels
• Injections: 
  • Intravenous (IV) injection or infusion into a vein
  • Intraventricular infusion into the ventricular space in the brain
  • Subcutaneous (SC) injection into the tissue under the skin
  • Intramuscular (IM), or

Other metabolic and endocrine drugs may be used in the treatment of conditions that include:

• Becaplermin:
  • Lower extremity diabetic neuropathic ulcers
• Betaine:
  • Homocystinuria is an inherited metabolic disorder that can affect normal growth and development in childhood, leading to disorders including eye abnormalities, mental retardation, and osteoporosis
• Bromocriptine:
  • Hyperprolactinemia (high level of prolactin, a hormone that regulates menstruation, sperm production, and lactation) in children and adults
  • Hyperprolactinemia-associated dysfunctions in adults, including amenorrhea (absence of menstruation), galactorrhea (breast milk secretion in men, or women who are not pregnant), infertility, and hypogonadism (deficiency in the function of reproductive glands, ovaries, and testes)
  • Parkinson’s disease (a progressive neurological disease)
  • Acromegaly (excessive growth of bones in the face, hands, and feet due to high levels of growth hormone in adulthood)
  • Type 2 diabetes mellitus
  • Neuroleptic malignant syndrome (off-label), a rare life-threatening reaction to antipsychotic (neuroleptic) drugs
• Carnitine (levocarnitine):
  • Carnitine deficiency
  • End-stage renal disease requiring hemodialysis
  • The orphan designation includes the following conditions:
  • Valproic acid overdose
  • Myopathy (muscle disorder)
  • Rett syndrome (a progressive neurodevelopmental disorder that primarily affects girls)
• Cerliponase alfa:
  • Infantile neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease, which is a group of diseases that primarily affects the nervous system.
• Cysteamine:
  • Nephropathic cystinosis is a genetic disorder that causes the accumulation of cystine, a leftover amino acid from metabolism, which crystallizes and damages organs including the kidneys
  • Fanconi syndrome in children is a condition in which the kidneys are unable to reabsorb electrolytes and many other substances (off-label)
  • Orphan designation for cysteamine use includes the following:
    • Cystic fibrosis, is a condition that affects cells that produce mucus and fluids, making the mucus and other fluids thick and sticky that clog up ducts and passages
    • Huntington’s disease, a rare inherited disease that causes progressive degeneration of nerve cells
    • Batten disease
    • Pancreatic cancer 
• Dextrose (glucose):
  • Hypoglycemia (high glucose level in blood)
• Dichlorphenamide:
  • Primary hyperkalemic periodic paralysis, primary hypokalemic periodic paralysis, and related variants
• Eliglustat:
  • Gaucher disease is a metabolic disorder that causes the accumulation of certain fats in cells, especially in bone marrow, liver, and spleen
• Nitisinone:
  • Hereditary tyrosinemia type 1, a genetic disorder that causes elevated levels of the amino acid tyrosine, the building block of most proteins
  • Alkaptonuria (orphan designation), is a genetic disorder that causes an inability to break up amino acids tyrosine and phenylalanine, resulting in black discoloration of urine and connective tissue
• Pegvisomant:
  • Acromegaly
• Triheptanoin:
  • Long-chain fatty acid oxidation disorders (LC-FAOD), a group of disorders that prevent the breakdown of fatty acids into energy
• Vestronidase alfa-vjbk:
  • Mucopolysaccharidosis VII (MPS VII or Sly syndrome) is a rare progressive metabolic disorder with enzyme deficiencies that lead to multisystem tissue and organ damage

Side effects of other metabolic and endocrine drugs vary with each drug. A few of the most common side effects may include:

• Becaplermin:
  • Erythema (redness)
  • Skin rash
  • Pain and burning at the application site
  • Skin ulceration
  • Ulcer infection and tunneling of ulcer
• Betaine:
  • Body odor
  • Diarrhea
  • Gastrointestinal distress
  • Anorexia (loss of appetite)
  • Nausea
  • Dysgeusia (taste disorder)
  • Alopecia (hair loss)
  • Dental disorders and glossitis (tongue inflammation)
  • Hives
• Bromocriptine:
  • Nausea
  • Rhinitis (inflammation of the nasal passage)
  • Headache
  • Asthenia (weakness)
  • Fatigue
  • Dizziness
  • Constipation
• Carnitine (levocarnitine):
  • Gastrointestinal distress and vomiting
  • Hypertension (high blood pressure)
  • Headache and fever
  • Dizziness and vertigo
  • Palpitations and tachycardia (rapid heartbeat)
  • Hypercalcemia (high level of calcium in the blood)
  • Anemia (low red blood cell count)
• Cerliponase alfa:
  • Pyrexia (fever)
  • Abnormal ECG readings
  • Decrease or increase in cerebrospinal fluid (CSF) proteins
  • Vomiting
  • Seizures
  • Hypersensitivity
  • Hematoma (pooling and clotting of blood outside a blood vessel)
• Cysteamine:
  • Nausea
  • Diarrhea/gastroenteritis/abdominal pain
  • Conjunctivitis (inflammation of the conjunctiva, the membrane over the whites of the eye and inner eyelid surfaces)
  • Influenza
  • Nasopharyngitis (inflammation of nose and throat)
  • Ear infection
  • Upper respiratory tract infection
• Dextrose (glucose):
  • Hyperosmolarity (high concentration of glucose, salt, and other substances in the blood)
  • Edema (swelling from excess fluid)/pulmonary edema
  • Venous thrombosis (blood clot blocking a vein) and phlebitis (venous inflammation)
  • Tachypnea (abnormally rapid breathing)
  • Hypervolemia (too much fluid in the blood)
  • Mental confusion, unconsciousness
  • Diarrhea and polydipsia (extreme thirst)
• Dichlorphenamide:
  • Paresthesia/hypoesthesia (abnormal/reduced skin sensation) 
  • Cognitive disorder
  • Dysgeusia (taste disorder)
  • Confusion
  • Headache
  • Lethargy and fatigue
  • Muscle spasms
• Eliglustat:
  • Arthralgia (joint pain)
  • Headache
  • Fatigue
  • Nausea
  • Diarrhea
  • Back pain
  • Pain in extremities
• Nitisinone:
  • Liver transplantation
  • Elevated tyrosine levels
  • Liver failure
  • Malignant hepatic neoplasm (liver tumor)
  • Leukopenia (low count of leukocytes, a type of white blood cell)
  • Thrombocytopenia (low platelet count in the blood)
  • Eye disorders such as corneal opacity or conjunctivitis
• Pegvisomant:
  • Injection site pain and reactions
  • Diarrhea
  • Nausea
  • Flu syndrome
  • Back pain
  • Chest pain
  • Dizziness
• Triheptanoin:
  • Abdominal pain
  • Diarrhea
  • Nausea
  • Vomiting
• Vestronidase alfa-vjbk:
  • Extravasation (leakage of fluid outside the blood vessel)
  • Diarrhea
  • Rash and pruritus (itching)
  • Infusion site swelling
  • Peripheral swelling
  • Anaphylaxis (serious allergic reaction)

Information contained herein is not intended to cover all possible side effects, precautions, warnings, drug interactions, allergic reactions, or adverse effects. Check with your doctor or pharmacist to make sure these products do not cause any harm when you take them along with other medicines. Never stop taking your medication and never change your dose or frequency without consulting your doctor.

Generic and brand names of some of the other metabolic and endocrine drugs include:

• becaplermin
• betaine
• Brineura
• bromocriptine
• carnitine
• Carnitor
• Cerdelga
• cerliponase alfa
• Cycloset
• Cystadane
• Cystagon
• cysteamine
• D50W
• dextrose
• DGlucose
• dichlorphenamide
• Dojolvi
• eliglustat
• glucose
• Keveyis
• levocarnitine
• Mepsevii
• nitisinone
• Nityr
• Orfadin
• Parlodel
• pegvisomant
• Procysbi
• recombinant human tripeptidyl peptidase 1 (rhtpp1)
• Regranex Gel
• Somavert
• triheptanoin
• vestronidase alfa-vjbk