The eight new drugs listed below probably won’t make any television commercials, but they will make a world of difference to those who suffer from these rare diseases and disorders.
Television commercials these days are rife with ads marketing the next great pharmaceutical breakthrough to treat arthritis, diabetes and even cancer. However, pharmaceutical companies aren’t just focused on treating the “big” diseases. They also spend their financial and intellectual capital creating treatments for diseases that are far less known to the wider public. The eight new drugs listed below probably won’t make any television commercials, but they will make a world of difference to those who suffer from these rare diseases and disorders.
- Sympazan (clobazam) Oral Film
Date of Approval: November 1, 2018
Company: Aquestive Therapeutics, Inc.
Treatment for: Lennox-Gastaut Syndrome
Lennox-Gastaut Syndrome is a severe form of childhood epilepsy that causes several different types of seizures. It is resistant to many typical antiseizure medications, which makes it difficult to treat. Aquestive Therapeutics used their PharmFilm technology to create a film version of clobazam. The use of film rather than tablets or liquid has the potential to allow a faster response that can combat the resistance of Lennox-Gestaut Syndrome. Sympazan also provides alternate delivery system for those who cannot take clobazam in other formats. The film resembles a postage stamp and can be taken without water.
- Firdapse (amifampridine phosphate) Tablets
Date of Approval: November 28, 2018
Company: Catalyst Pharmaceuticals, Inc.
Treatment for: Lambert-Eaton Myasthenic Syndrome
Lambert-Eaton Myasthenic Syndrome is a rare autoimmune disorder in which the nerves do not communicate effectively with the muscles, causing growing muscle weakness. It can present with or without small cell lung cancer. There is no cure. Treatment focuses on management of the disease. Firdapse (amifampridine phosphate) was created by Catalyst Pharmaceuticals as an oral medication for patients 17 years and older. Firdapse is a modification of 3,4-diaminopyridine (3,4-DAP) that is considered more reliable than 3,4-DAP. Amifampridine phosphate is reported to be the safest and most effective medication for the treatment of Lambert Eaton Myasthenic Syndrome. Catalyst Pharmaceuticals is currently in litigation with the FDA over its approval, Ruzurgi (see #5) of a potential competitor in the market.
- Egaten (triclabendazole) Tablets
Date of Approval: February 13, 2019
Company: Novartis
Treatment for: Fascioliasis
Fascioliasis is an infectious disease caused by liver flukes that live on water plants primarily in Europe and Asia. Humans are infected after eating the plants or drinking contaminated water. Once ingested, the liver flukes block the bile passages in the liver. Egaten (triclabendazole) is the only FDA approved medication for the treatment of fascioliasis and the only fascioliasis medication recommended by the World Health Organization.
- Dengvaxia (dengue tetravalent vaccine, live) Injection
Date of Approval: May 1, 2019
Company: Sanofi Pasteur, Inc.
Treatment for: Prevention of Dengue Disease
Dengue Disease has been in the news recently due to a recent outbreak in India. It is transmitted via bites from infected mosquitos. The CDC estimates that dengue disease affects about 400 million people per year and, of those, approximately 22,000 per year die of the disease. Dengvaxia is administered as three separate injections over a twelve-month period. It is designed for people who have already suffered one bout of dengue disease, since subsequent dengue infections are more severe.
- Ruzurgi (amifampridine) Tablets
Date of Approval: May 6, 2019
Company: Jacobus Pharmaceutical Company, Inc.
Treatment for: Lambert-Eaton Myasthenic Syndrome
Like Firdapse (see #2), Ruzurgi is designed to treat Lambert-Eaton Myasthenic Syndrome. However, there are some significant differences between the two drugs. Firdapse is designed for patients 17 years and older, while Ruzurgi is designed for pediatric patients of the disease. Both drugs use a base of amifampridine, but Firdapse uses amifampridine phosphate, which allows it to be stored at room temperature. The drugs were released with six months of one another, and each is considered about off brand use of the other infringing on their customer base.
- Thiola EC (tiopronin) Delayed-Release Tablets
Date of Approval: June 28, 2019
Company: Retrophin, Inc.
Treatment for: Cystinuria
Cystinuria is a disorder inherited as an autosomal recessive genetic trait. The disorder causes the individual to excrete such abnormally high levels of cystine into the urine that it cannot be dissolved. This causes kidney pain, hematuria, ureters, and/or urinary tract infections. Thiola EC was created by Retrophin, a company that focuses on creating drugs for rare diseases. Thiola EC is an enteric-coated formulation of Thiola (tiopronin), which breaks the disulphide bond of cystine and then binds the cystine monomers with tiopronin to make it more water soluble. The new formulation is delayed-release, which will make it more convenient for patients of this disorder.
- Jynneos (smallpox and monkeypox vaccine) Injection
Date of Approval: September 24, 2019
Company: Bavarian Nordic
Treatment for: Prevention of Smallpox; Prevention of Monkeypox
Almost everyone has heard of smallpox. Monkeypox, however, is less well known. It was first discovered in monkey colonies, hence the name. The cause of monkeypox is unknown. It causes a rash that turns to lesions or pustules across the body. Jynneos is the first vaccine to be approved to treat monkeypox. It is also the only non-replicating vaccine to be approved for the treatment of smallpox. Jynneos is a live attenuated vaccine that is administered via injection. It is approved for use in patients over 18 years of age who are determined to be at risk for either smallpox or monkeypox infection.
- Scenesse (afamelanotide) Implant
Date of Approval: October 8, 2019
Company: Clinuvel Pharmaceuticals Ltd.
Treatment for: Prevention of Phototoxicity in Erythropoietic Protoporphyria
Erythropoietic Protoporphyria is an inherited disorder that is caused by a lack of the enzyme ferrochelatase. This causes excess levels of protoporphyrin in the bone marrow, plasma, and red blood cells. The primary symptom of this disorder is severe pain when exposed to sunlight and some artificial light, a condition called phototoxicity. Scenesse (afamelanotide) is a synthetic form of a naturally occurring peptide that is released by skin cells as a response to ultraviolet rays. Treatment requires implanting short white rods containing afameanotide under the skin. This will cause the patient to produce eumelanin, which ca aid in increasing the patient’s ability to tolerate sun exposure.
