2018 Drug Approvals

The FDA approved 59 investigational drugs to be released to distribution in 2018, beating the 2017 total of 46. The number of approvals in 2018 also sets a record for the number of new drug approvals in a single year. The previous highest annual total was 53 in 1996. 
​
Almost 3/4 of the drugs (43) had priority reviews. Almost half of the priority reviews were filed as orphan drugs (34), while 58% were for rare diseases, 41% had Fast Track status and almost a quarter were designated as Breakthrough Therapies (14). Through its work in streamlining and optimizing drug reviews, 71% of drugs were first approved in the U.S. All drugs were approved by or before their PDUFA dates. 
 
The FDA was not the only one that increased the number of new drugs approved in 2018. The European Medicine Agency approved 42 new drugs last year, which is up from 35 in 2017.  This included 21 drugs that were designated Orphan Drugs. As in the U.S., the largest therapeutic group were the 23 oncology drugs approved.
 
Congress passed Right-To-Try legislation in the spring of 2018. Formal guidelines have not been announced, but ERC-1671 (Gliovacfrom Epitopoietic Research Corp)became the first right to try drug in the fall.
 
Notable approvals in 2018
 
A total of 14 infectious disease drugs that included:

• Baloxavir marboxil(Xofluza), was approved as a single dose influenza treatment that reduced flu symptoms similar to 5-days of oseltamivir.
• Three drugs for MDR bacterial infections
  • Eravacycline (Xerava) was approved for the treatment of complicated intra-abdominal infections.
  • Omadacycline (Nuzyra) was approved for the treatment of community-acquired pneumonia and acute skin and skin structure infections.
  • Plazomicin (Zemdri) was approved for the treatment of complicated urinary tract infections and is the first aminoglycoside approved since amikacin.
• Five drugs or new combinations for the treatment of HIV
  • Bictegravir, emtricitabine, tenofovir alafenamide (Biktarvy) 
  • Doravirine (Pifeltro) 
  • Doravirine, lamivudine and tenofovir disoproxil fumarate (Delstrigo) 
  • Ibalizumab (Trogarzo) was approved for multi-drug resistant HIV 
  • darunavir, cobicistat, emtricitabine, and tenofovir alafenamide (Symtuza)

A total of seven neurology drugs that included:

• Three CGRP for migraine prophylaxis 
  • Erenumab (Aimovig) 
  • Fremanezumab (Ajovy)
  • Galcanezumab(Emgality)
• The first cannabidiol (Epidiolex) was approved for the the treatment of seizures associated with Lennox-Gastaut syndrome or Dravet syndrome.

A total of six drugs for genetic diseases that included:

• Two drugs for the treatment of the polyneuropathy of hereditary transthyretin mediated amyloidosis. 
  • Patisiran(Onpattro)
  • Inotersen (Tegsedi)
• Migalastat (Galafold) for the treatment of Fabry disease caused by a genetic mutation
• Burosumab (Crysvita) for the treatment of X-linked hypophosphatemia
• Elapegademase(Revcovi) for the treatment of adenosine deaminase severe combined immune deficiency
• Lanadelumab (Takhzyro) for the treatment of hereditary angioedema

A total of 16 oncology drugs were approved including:

• More treatments for hematologic cancers than for solid tumors.
• Several drugs for the treatment of genetically specified tumors were approved.
  • Binimetinib (Mektovi) + Encorafenib(Braftovi) for the treatment of unresectable or metastatic melanoma with a BRAFV600E or BRAFV600K mutation 
  • Ivosidenib(Tibsovo) for the treatment of relapsed or refractory acute myeloid leukemia with an IDH1 mutation
  • Dacomitinib(Vizimpro) for the treatment of EGFR-mutated non-small cell lung cancer
  • Lorlatinib (Lorbrena) for the treatment of ALK-positive NSCLC
  • Talazoparib (Talzenna) for the treatment of BRCA-mutated, HER2-negative breast cancer
  • Larotrectinib (Vitrakvi) for the treatment of tumors feature a neurotrophic receptor tyrosine kinase gene fusion
  • Gilteritinib(Xospata) for the treatment of acute myeloid leukemia with an FLT3mutation

Specialty Pharmacy Times listed their top specialty pharmaceuticals approved in 2018

• Erenumab (Aimovig) for tprophylaxis of migraine headaches 
• Lanadelumab (Takhzyro) for the treatment of hereditary angioedema
• Baricitinib (Olumiant) for the treatment of rheumatoid arthritis
• Cannabidiol (Epidiolex) for the treatment of Lennox-Gastaut syndrome and Dravet syndrome
• Tildrakizumab(Ilumya) for the treatment of plaque psoriasis
• Tofacitinib (Xeljanz) for the treatment of active ulcerative colitis

Miscellaneous drug approvals

• ElagolixOrilissa) for the treatment of pain associated with endometriosis
• Coagulation factor Xa (Andexxa) for reversal of factor Xa inhibitors
• Tezacaftor/ivacaftor (Symdeko) for the treatment of cystic fibrosis. This two drug combination will become a three drug combination in the next 1-3 years, when either VX-659 and VX-445 (correctors) are added to the two drug combination.

Resources
Medscape's Review of New Drug Approvals
 
The FDA's 2018 Drug Therapy Approvals
 
The FDA's List of 2018 Drug Approvals by Medical Specialty
 
PM Live's Review of European 2018 Drug Approvals 
 
Specialty Pharmacy Time's Review of Top Ten Specialty Pharmaceutical Approvals