(January 7, 2018)
The FDA approved 46 new chemical drugs and three important biological drugs in 2017 after only approving 22 chemical drugs in 2016. The four drugs below were fast-tracked for approval under "priority review" status and also represent "first-in-class" innovation for the drug type. The first two are for extremely rare diseases that afflict children and received rare FDA pediatric disease priority review. The second two treat rare forms of leukemia, including a break-through gene therapy (Kymriah) that is only approved for use in children and young adults up to age 25.
1. Brineura (cerliponase alfa) |
|
Type of drug: |
Biological |
|
Administered:
|
Injection
|
|
Approved for:
|
CLN2, Battern Disease
|
|
Battern disease is a rare fatal inherited brain disease;
children born with the disease start showing symptoms at age 2
and many lose the ability to walk and talk at around age 6. Most
die by their teens. This drug slows the progression of loss of
ambulation so it helps the child to be able to walk longer. It
is an enzyme replacement therapy. Only 20 children are born with
the disease in the U.S. each year.
|
|
|
Patient age:
|
pediatric patients age 3+
|
|
Company:
|
Biomarin Pharmaceutical
|
|
Estimated Cost:
|
$702,000 per year before any discounts
|
|
2. Mepsevii
(vestronidase alfa-vbjk)
|
|
|
Type of drug:
|
Biological
|
|
Administered:
|
Injection
|
|
Approved for:
|
Sly Syndrome
|
|
Treats an extremely rare, inherited metabolic condition called
mucopolysaccharidosis type VII (MPS VII), (Sly syndrome). There
are fewer than 100 cases reported in the US and only about 200
cases in the developed world. The disease is progressive and
affects most tissues and organs due to a defective gene.
Mepsevii is an enzyme replacement therapy. The drug has a Black
Box Warning for anaphylaxis risk.
|
|
|
Patient age:
|
pediatric and adult patients
|
|
Company:
|
Ultragenyx Pharmaceutical Inc.
|
|
Estimated Cost:
|
$550,000 per year before any discounts
|
|
3. Idhifa
(enasidenib)
|
|
|
Type of drug:
|
Chemical
|
|
Administered:
|
Tablet; oral
|
|
Approved for:
|
Relapsed or refractory acute myeloid leukemia (AML)
|
|
Approved under orphan drug status. Contains black box warning
for differentiation syndrome which can be fatal if not treated.
Symptoms of the syndrome may include fever and acute respiratory
distress.
|
|
|
Patient age:
|
adult patients with IDH2 mutation
|
|
Company:
|
Celgene Corporation
|
|
Estimated Cost:
|
$24,872 per month before discounts.
|
|
|
|
|
4. Kymriah
(tisagenlecleucel)
|
|
|
Type of drug:
|
Biological
|
|
Administered:
|
Infusion
|
|
Approved for:
|
Acute Lymphoblastic Leukemia (ALL)
|
|
For patients with ALL that is in second or later relapse. This
drug is a break-through drug in that it is the first
FDA-approved gene therapy.The drug is created from the patient's
own T-cells, which are extracted, genetically modified, and
infused back. It carries a dangerous risk of cytokine release
syndrome (CRS), which is a life-threatening autoimmune reaction.
|
|
|
Patient age:
|
patients age 3 - 25
|
|
Company:
|
Novartis Pharmaceutical
|
|
Estimated Cost:
|
$475,000 per treatment.
|
|
|
Interesting "outcomes based" pricing possible; the patient only
pays if they go into remission within three months of
treatment.Only one treatment required.
|
Tables compiled by AskaPatient.
Cost estimates from Fierce Biotech website.
Cost estimates from Fierce Biotech website.