First-in-class medication

Last updated

A first-in-class medication is a prototype drug that uses a "new and unique mechanism of action" to treat a particular medical condition. [1] While the Food and Drug Administration's Center for Drug Evaluation and Research tracks first-in-class medications and reports on them annually, first-in-class is not considered a regulatory category. Although many first-in-class medications qualify as breakthrough therapies, Regenerative Medicine Advanced Therapies and/or orphan drugs, first-in-class status itself has no regulatory effect.

Contents

Examples

DrugClassTargeted conditionsYear approved

(FDA)

Year approved

(EMA)

Inotuzumab ozogamicin

(Besponsa)

Anti-CD22 monoclonal antibody-drug conjugate Relapsed or refractory B cell precursor acute lymphoblastic leukemia [2] 2017
Tagraxofusp

(Elzonris)

 Interleukin 3-diphtheria toxin fusion protein targeting plasmacytoid dendritic cells Blastic plasmacytoid dendritic cell neoplasm 20182021
Midostaurin

(Rydapt)

Multi-target tyrosine kinase inhibitor not inhibited by the D816V cKit mutation Systemic mastocytosis, myelodysplastic syndrome, acute myeloid leukemia [3] 20172017 [4]
Teprotumumab

(Tepezza)

Anti-IGF-1R monoclonal antibody Graves' ophthalmopathy [5] 2020
Romosozumab

(Evenity)

Anti-sclerostin monoclonal antibody Osteoporosis [6] 20192019
Ocrelizumab

(Ocrevus)

Anti-CD20 monoclonal antibody Multiple sclerosis [7] 20172018
Ivosidenib

(Tibsovo)

Small molecule inhibitor of isocitrate dehydrogenase 1 Acute myeloid leukemia, cholangiocarcinoma [8] 20182023
Bempedoic acid

(Nexletol)

 Adenosine triphosphate-citrate lyase inhibitor Hypercholesterolemia [9] 20202020
Tafamidis

(Vyndaqel, Vyndamax)

 Transthyretin chaperone (stabilizer) Familial amyloid polyneuropathy and other transthyretin amyloidoses [10] 20112019
Voxelotor

(Oxbryta)

 Hemoglobin oxygen affinity modulator Sickle cell disease [11] 2019
Lonafarnib

(Zokinvy)

 Farnesyltransferase inhibitor Hutchinson-Gilford progeria syndrome [12] 20202022
Dupilumab

(Dupixent)

 Interleukin-4 receptor alpha subunit inhibitor Asthma, atopic dermatitis, allergic diseases [13] 2017
Lasmiditan

(Reyvow)

Selective 5-HT1F serotonin receptor agonist Migraine [14] 20192022
Tazemetostat

(Tazverik)

Selective EZH2 inhibitor Epithelioid sarcoma [15] 2020
Tirzepatide

(Mounjaro)

 GLP-1 and GIP activatoradult type 2 diabetes [16] 20222022

Controversy

Safety

By definition, a first-in-class drug does not have the safety evidence from analogous products that not-first-in-class drugs would have. However, a study investigating recalls and warnings in relation to first-in-class drugs approved between 1997 and 2012 by Health Canada has found that first-in-class drugs actually have a more favourable benefit-to-harm ratio. [17]

Economics

First-in-class drugs are often seen as commercially more attractive as they may tap into a market segment that has hitherto been underserved, but this may be illusory. [18] In fact, most blockbuster drugs (drugs with annual sales revenues exceeding US$1,000,000,000) were not first-in-class drugs. [18] The economic potential of a first-in-class drug, which is typically priced higher than later drugs in the same class, has been largely declining due to efforts by health insurers to restrict what specialty drugs are covered and prevent incumbency advantages. [19]

Costs

A lower number of available therapeutic options correlates with higher prices. [20] In addition, many first-in-class medications are specialty drugs and orphan drugs, [21] which means that manufacturers have to recoup development costs from a smaller market. [22] This raises ethical questions about the sustainability of the high prices on these costs. [23] [24]

Related Research Articles

<span class="mw-page-title-main">Pentamidine</span> Medical antimicrobial drug

Pentamidine is an antimicrobial medication used to treat African trypanosomiasis, leishmaniasis, Balamuthia infections, babesiosis, and to prevent and treat pneumocystis pneumonia (PCP) in people with poor immune function. In African trypanosomiasis it is used for early disease before central nervous system involvement, as a second line option to suramin. It is an option for both visceral leishmaniasis and cutaneous leishmaniasis. Pentamidine can be given by injection into a vein or muscle or by inhalation.

Prescription drug list prices in the United States continually are among the highest in the world. The high cost of prescription drugs became a major topic of discussion in the 21st century, leading up to the American health care reform debate of 2009, and received renewed attention in 2015. One major reason for high prescription drug prices in the United States relative to other countries is the inability of government-granted monopolies in the American health care sector to use their bargaining power to negotiate lower prices, and the American payer ends up subsidizing the world's R&D spending on drugs.

<span class="mw-page-title-main">Pravastatin</span> Cholesterol lowering medication in the statin class

Pravastatin, sold under the brand name Pravachol among others, is a statin medication, used for preventing cardiovascular disease in those at high risk and treating abnormal lipids. It is suggested to be used together with diet changes, exercise, and weight loss. It is taken by mouth.

<span class="mw-page-title-main">Spinocerebellar ataxia</span> Medical condition

Spinocerebellar ataxia (SCA) is a progressive, degenerative, genetic disease with multiple types, each of which could be considered a neurological condition in its own right. An estimated 150,000 people in the United States have a diagnosis of spinocerebellar ataxia at any given time. SCA is hereditary, progressive, degenerative, and often fatal. There is no known effective treatment or cure. SCA can affect anyone of any age. The disease is caused by either a recessive or dominant gene. In many cases people are not aware that they carry a relevant gene until they have children who begin to show signs of having the disorder.

<span class="mw-page-title-main">Medicare Part D</span> United States prescription drug benefit for the elderly and disabled

Medicare Part D, also called the Medicare prescription drug benefit, is an optional United States federal-government program to help Medicare beneficiaries pay for self-administered prescription drugs. Part D was enacted as part of the Medicare Modernization Act of 2003 and went into effect on January 1, 2006. Under the program, drug benefits are provided by private insurance plans that receive premiums from both enrollees and the government. Part D plans typically pay most of the cost for prescriptions filled by their enrollees. However, plans are later reimbursed for much of this cost through rebates paid by manufacturers and pharmacies.

Ethinylestradiol/norethisterone acetate (EE/NETA), or ethinylestradiol/norethindrone acetate, is a combination of ethinylestradiol (EE) and norethisterone acetate (NETA) which is used as birth control and menopausal hormone therapy. EE is an estrogen, while norethisterone acetate (NETA) is a progestin. It is taken by mouth. Some preparations of EE/NETA used in birth control additionally contain an iron supplement in the form of ferrous fumarate.

Atenolol/chlorthalidone, also known as co-tenidone, is a combination medication used to treat high blood pressure. It is made up of atenolol, a beta-blocker and chlortalidone, a diuretic. It is not recommended as an initial treatment but may be used in those who are taking atenolol and chlortalidone individually. It is taken by mouth.

<span class="mw-page-title-main">Repaglinide</span> Chemical compound

Repaglinide is an antidiabetic drug in the class of medications known as meglitinides, and was invented in 1983. Repaglinide is a medication used in addition to diet and exercise for blood sugar control in type 2 diabetes. The mechanism of action of repaglinide involves promoting insulin release from β-islet cells of the pancreas; like other antidiabetic drugs, a main side effect concern is hypoglycemia. It is sold by Novo Nordisk under the name of Prandin in the United States, Gluconorm in Canada, Surepost in Japan, Repaglinide in Egypt, and Novonorm elsewhere. In Japan it is produced by Dainippon Sumitomo Pharma.

<span class="mw-page-title-main">Emtricitabine/tenofovir</span> Drug combination for HIV/AIDS prophylaxis and treatment

Emtricitabine/tenofovir, sold under the brand name Truvada among others, is a fixed-dose combination antiretroviral medication used to treat and prevent HIV/AIDS. It contains the antiretroviral medications emtricitabine and tenofovir disoproxil. For treatment, it must be used in combination with other antiretroviral medications. For prevention before exposure, in those who are at high risk, it is recommended along with safer sex practices. It does not cure HIV/AIDS. Emtricitabine/tenofovir is taken by mouth.

<span class="mw-page-title-main">Medication package insert</span> Document included in a package of medicine

A package insert is a document included in the package of a medication that provides information about that drug and its use. For prescription medications, the insert is technical, providing information for medical professionals about how to prescribe the drug. Package inserts for prescription drugs often include a separate document called a "patient package insert" with information written in plain language intended for the end-user—the person who will take the drug or give the drug to another person, such as a minor. Inserts for over-the-counter medications are also written plainly.

<span class="mw-page-title-main">Pegaspargase</span> Pharmaceutical drug

Pegaspargase, sold under the brand name Oncaspar, is a medication used in the treatment of acute lymphoblastic leukemia (ALL). Often it is used together with anthracycline, vincristine, and corticosteroids. Pegaspargase can be administered either via an intravenous infusion or a intramuscular injection.

In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis is the assessment that a particular medical condition is present while an indication is a reason for use. The opposite of an indication is a contraindication, a reason to withhold a certain medical treatment because the risks of treatment clearly outweigh the benefits.

Neomycin/polymyxin B/hydrocortisone, sold under the brand Otosporin among others, is a medication used to treat otitis externa and certain eye disorders. It consists of the antibiotics neomycin and polymyxin B, and the steroid hydrocortisone. It is used as an ear drop or eye drop.

<span class="mw-page-title-main">Acetylleucine</span> Chemical compound

Acetylleucine is a modified amino acid used in the treatment of vertigo and cerebellar ataxia.

Pitolisant, sold under the brand name Wakix among others, is a medication used for the treatment of excessive daytime sleepiness in adults with narcolepsy. It is a histamine 3 (H3) receptor antagonist/inverse agonist (an antihistamine drug specific to that kind of receptors). It represents the first commercially available medication in its class, so that the US Food and Drug Administration (FDA) declares it a first-in-class medication. Pitolisant enhances the activity of histaminergic neurons in the brain that function to improve a person's wakefulness.

<span class="mw-page-title-main">Phentermine/topiramate</span> Obesity medication

Phentermine/topiramate, sold under the brand name Qsymia, is a combination drug of phentermine and topiramate used to treat obesity. It is used together with dietary changes and exercise. If less than 3% weight loss is seen after 3 months it is recommended the medication be stopped. The weight loss is modest. Effects on heart related health problems or death is unclear.

<span class="mw-page-title-main">Efinaconazole</span> Chemical compound

Efinaconazole, sold under the brand name Jublia among others, is a triazole antifungal medication. It is approved for use in the United States, Canada, and Japan as a 10% topical solution for the treatment of onychomycosis. Efinaconazole acts as a 14α-demethylase inhibitor.

An analgesic adjuvant is a medication that is typically used for indications other than pain control but provides control of pain (analgesia) in some painful diseases. This is often part of multimodal analgesia, where one of the intentions is to minimize the need for opioids.

<span class="mw-page-title-main">Ezetimibe/rosuvastatin</span> Cholesterol medication

Ezetimibe/rosuvastatin, sold under the brand name Ridutrin among others, is a combination medication used to treat high cholesterol. In some countries it is sold as a kit or a pack containing two distinct pills.

<span class="mw-page-title-main">Oteseconazole</span> Chemical compound

Oteseconazole, a novel orally bioavailable and selective inhibitor of fungal cytochrome P450 enzyme 51 (CYP51), has shown promising efficacy in the treatment of recurrent vulvovaginal candidiasis (RVVC) in patients.

References

  1. ↑ Lanthier, Michael; Miller, Kathleen L.; Nardinelli, Clark; Woodcock, Janet (2013-08-01). "An Improved Approach To Measuring Drug Innovation Finds Steady Rates Of First-In-Class Pharmaceuticals, 1987–2011". Health Affairs. 32 (8): 1433–1439. doi:10.1377/hlthaff.2012.0541. ISSN   0278-2715. PMID   23918488.[ permanent dead link ]
  2. ↑ "BESPONSA 1 mg powder for concentrate for solution for infusion - Summary of Product Characteristics (SmPC) - (emc)". www.medicines.org.uk. Retrieved 2021-07-01.
  3. ↑ "Rydapt: Uses, Dosage, Side Effects, Warnings". Drugs.com. Retrieved 2021-07-01.
  4. ↑ "Rydapt | European Medicines Agency". www.ema.europa.eu. Retrieved 2024-01-16.
  5. ↑ Office of the Commissioner (2020-03-24). "FDA approves first treatment for thyroid eye disease". FDA. Retrieved 2021-07-01.
  6. ↑ Center for Drug Evaluation and Research (2020-01-24). "New Drug Therapy Approvals 2019". FDA.
  7. ↑ "Ocrevus (ocrelizumab) Injection". www.accessdata.fda.gov. Retrieved 2021-07-01.
  8. ↑ "Search Orphan Drug Designations and Approvals". www.accessdata.fda.gov. Retrieved 2021-07-01.
  9. ↑ "DailyMed - NEXLETOL- bempedoic acid tablet, film coated". dailymed.nlm.nih.gov. Retrieved 2021-07-01.
  10. ↑ "Tafamidis Monograph for Professionals". Drugs.com. Retrieved 2021-07-01.
  11. ↑ "DailyMed - OXBRYTA- voxelotor tablet, film coated". dailymed.nlm.nih.gov. Retrieved 2021-07-01.
  12. ↑ "DailyMed - ZOKINVY- lonafarnib capsule". dailymed.nlm.nih.gov. Retrieved 2021-07-01.
  13. ↑ "DailyMed - DUPIXENT- dupilumab injection, solution". dailymed.nlm.nih.gov. Retrieved 2021-07-01.
  14. ↑ "DailyMed - Search Results for Lasmiditan". dailymed.nlm.nih.gov. Retrieved 2021-07-01.
  15. ↑ "Tazemetostat Monograph for Professionals". Drugs.com. Retrieved 2021-07-01.
  16. ↑ "FDA Approves Novel, Dual-Targeted Treatment for Type 2 Diabetes". fda.gov. 13 May 2022. Retrieved 2022-06-30.
  17. ↑ Lexchin, Joel (November 2016). "How Safe and Innovative Are First-in-Class Drugs Approved by Health Canada: A Cohort Study". Healthcare Policy. 12 (2): 65–75. ISSN   1715-6572. PMC   5221712 . PMID   28032825.
  18. 1 2 Schulze, Ulrik; Ringel, Michael (2013-06-01). "What matters most in commercial success: first-in-class or best-in-class?". Nature Reviews Drug Discovery. 12 (6): 419–420. doi:10.1038/nrd4035. ISSN   1474-1784. PMID   23722339. S2CID   32258945.
  19. ↑ Longman, Roger (20 July 2015). "The Shrinking Value of Best-in-Class and First-in-Class Drugs". In Vivo by Informa Pharma Intelligence. Retrieved 1 July 2021.
  20. ↑ Kwa, Michael C.; Tegtmeyer, Kyle; Welty, Leah J.; Raney, Sam G.; Luke, Markham C.; Xu, Shuai; Kong, Betty (2020-10-01). "The relationship between the number of available therapeutic options and government payer (medicare part D) spending on topical drug products". Archives of Dermatological Research. 312 (8): 559–565. doi:10.1007/s00403-020-02042-9. ISSN   1432-069X. PMID   32055932. S2CID   211111984.
  21. ↑ Chambers, James D.; Thorat, Teja; Wilkinson, Colby L.; Neumann, Peter J. (2017-08-01). "Drugs Cleared Through The FDA's Expedited Review Offer Greater Gains Than Drugs Approved By Conventional Process". Health Affairs. 36 (8): 1408–1415. doi: 10.1377/hlthaff.2016.1541 . ISSN   0278-2715. PMID   28784733.
  22. ↑ Lieberman, Steven M.; Ginsburg, Paul B.; Patel, Kavita K. (2020). "Balancing Lower U.S. Prescription Drug Prices And Innovation – Part 1 | Health Affairs Blog". Health Affairs Forefront. doi:10.1377/forefront.20201123.804451.
  23. ↑ Herper, Matthew (2019-12-23). "The debate over America's drug-pricing system is built on myths". STAT. Retrieved 2021-07-01.
  24. ↑ Greene, Jan (January 2017). "EpiPen Controversy Reveals Complexity Behind Drug Price Tags". Annals of Emergency Medicine. 69 (1): A16–A19. doi: 10.1016/j.annemergmed.2016.10.025 . ISSN   0196-0644.
This page is based on this Wikipedia article
Text is available under the CC BY-SA 4.0 license; additional terms may apply.
Images, videos and audio are available under their respective licenses.