A Recap on 2018: Record Number of Drugs Approved by the FDA for Rare Diseases

Each year the Food and Drug Administration approves dozens of new therapies to fulfill unmet needs for various patient populations. It’s just been published that in 2018 they approved a record breaking 59 novel drugs. Novel drugs are defined by the FDA as new molecular entities.

Perhaps even more noteworthy than the number of drugs approved last year, is the fact that the majority of these drugs were approved for the treatment of rare diseases. 34 drugs to be exact, were approved as new therapeutic options for rare populations.

It may be slow, but it’s clear we are making progress for rare communities.

History of approval

For the last 190 years the FDA has approved around the same number of drugs every year. Of course this has fluctuated some, but it has never been as high as 59. The average number of treatments receiving the FDA stamp of approval has been 33. In 2017 46 were approved, and in 2016 a mere 22 received approval.

The 59 drugs approved last year spanned a wide range of ailments. Some of these included migraines, chronic obstructive pulmonary disease, many different cancers, and traveler’s diarrhea.

Some Noteworthy Approvals from 2018

32% of the approved drugs in 2018 were given the designation of “first-in-class.” This classification is generally a good predictor of the impact a drug will have on their patient population. One of the 19 drugs given this title during 2018 was for the treatment of Fabry disease. This is a rare condition which results in the build up of fat in various parts of the body such as heart, the kidneys, and the blood vessels. The FDA gave approval to the very first oral medication, called Galafold, to be used for adults with the disease.

Another rare disease victory was Oxervate for the treatment of neurotrophic keratitis, a progressive eye condition which affects an estimated 65,000 individuals in the United States.

Epidiolex was also approved for the treatment of Lennox-Gastaut syndrome and Dravet syndrome, two rare forms of epilepsy. This approval was particularly noteworthy because it was the first drug ever granted approval by the FDA which contained a substance derived from marijuana.

A second drug approved for Dravet syndrome last year (for patients age 2 and older taking clobazam) was Diacomit, created by Biocodex.

Sezary syndrome, a type of non-Hodgkin’s lymphoma, also received an approval in 2018. The drug is called Poteligeo, an intravenous injection. It was specifically designed for patients who have previously had a systemic therapy.

One more noteworthy approval for the rare community was Onpattro, created for the treatment of the polyneuropathy which accompanies a form amyloidosis (hATTR). This drug was the first, and as of now is still the only, RNAi therapeutic to receive approval from the FDA.

Looking forward

There is always more research to be done for rare diseases, but these approvals showcase progress and provide hope to those living with rare conditions that more novel therapeutic creations are coming. Let’s keep our fingers crossed that we surpass this record of approvals in 2019.

You can read more about the 2018 FDA approvals here.


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