New Drugs Introduced in 2017: Part 3 Genetic Diseases, Hematology, and Liver Disease


This year, the FDA is on pace to approve about 38 new medications. Here is a look at 4 of those.

This year, the FDA is on pace to approve about 38 new medications. Additions to the drug market in 2017 cover a variety of diseases and ailments providing prescribers with more options and the ability to optimize drug therapy. Part 3 of this series covers new medications for genetic diseases, hematology, and liver disease.

Genetic Diseases

Brineura (cerliponase alfa)

The FDA approved cerliponase alfa on April 27, 2017, and it is the first approved treatment for any form of Batten disease, an extremely rare and fatal autosomal recessive neurodegenerative disorder that begins in childhood.1 It is a hydrolytic lysosomal N-terminal tripeptidyl peptidase (proenzyme) that, once activated, leads to the breakdown of lysosomal storage materials that otherwise would build up in patients with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2). Therefore, it is indicated to slow the loss of ambulation in symptomatic pediatric patients 3 and older with CLN2.2 CLN2 is an extremely rare and rapidly progressing brain disorder that affects an estimated 20 children in the United States each year (<1 in a million Americans/year).1,2 Cerliponase alfa is formulated as a solution for IV injection (150mg/5mL) and is administered by a physician to the cerebrospinal fluid (CSF) via a surgically implanted reservoir and catheter. The first dose should be given 5 to 7 days after implanting the reservoir/catheter. It is recommended to pretreat with antihistamines with or without antipyretics or corticosteroids 30 to 60 minutes prior to the start of infusion due to the adverse reactions listed below. The recommended dosage is 300mg (10 mL) administered as an IV infusion once every other week, followed by the infusion of 2 mL IV electrolytes, which are included in the administration kit, over about 4.5 hours.1,3 Contraindications to cerliponase alfa include acute IV access complications, such as leakage, device failure, or infection, and patients with ventriculoperitoneal shunts.1 The most common adverse effects (>8% compared with the placebo) include pyrexia, ECG abnormalities, increased/decreased CSF protein, vomiting, seizures, hypersensitivity reactions, hematoma, headache, irritability, pleocytosis, device-related infection, bradycardia, feeling jittery, and hypotension.1 Brineura™ costs about $27,000 per biweekly infusion. This results in a yearly cost of nearly $702,000 if no discounts or federal assistance programs are in place for the patient.2,4

Haegarda (C1 Esterase Inhibitor [human])

Haegarda is a new subcutaneous formulation of a medication that was initially approved in 2008 and approved for subcutaneous injection on June 22, 2017. This formulation allows for much easier home dosing by the patient or caregiver, compared with previous formulations, which were only intravenous.5,6 Haegarda is the first C1 esterase inhibitor (human) for subcutaneous injection approved for prevention of hereditary angioedema (HAE) attacks in adult and adolescent patients.5,7 C1 esterase inhibitor therapy in patients with C1 inhibitor deficiency, such as HAE, is thought to suppress contact system activation by inactivating plasma kallikrein and factor XIIa. Therefore, bradykinin production is prevented. Unregulated bradykinin production is thought to contribute to the increased vascular permeability and angioedema observed in HAE.5,6,7 Haegarda comes as freeze-dried powder for reconstitution in either a 2000-unit or 3000-unit vial and is recommended to be given as a subcutaneous injection of 60 units/kg every 3 or 4 days (twice per week).6,7 The most common adverse effects (>4% compared with the placebo), include injection site reaction, hypersensitivity, nasopharyngitis, and dizziness.7 Other warnings and precautions include severe hypersensitivity reaction requiring epinephrine and transmission of infectious agents due to it being made from human blood.7 Haegarda costs $2256 for the 2000 units of powder and $3384 for the 3000 units of powder.6

Click to read about Hereditary Angioedema: The Pharmacist's Role in Team-Based Patient Care.


Endari (L-glutamine oral powder)

Endari is a new formulation of glutamine that was approved on July 7, 2017. Endari is the first drug formulation approved in nearly 20 years for the treatment of sickle cell disease and is specifically indicated to reduce the severe complications of sickle cell disease in adult and pediatric patients 5 and older.8,9 Sickle cell disease is an inherited blood disorder that results in abnormally shaped red blood cells. This abnormal shape restricts blood flow in vessels limiting oxygen supply to different tissues. This can lead to severe pain and organ damage,10 In sickle cell disease, red blood cells are more vulnerable to oxidative damage than normal blood cells. L-glutamine’s activity in sickle cell disease comes from the fact that it is a biological precursor to nicotinamide adenine dinucleotide (NAD). NAD, and its reduced form, NADH, help regulate and prevent red blood cell oxidative damage by increasing the availability of reduced glutathione.11 Endari is supplied as a powder packet for oral administration (5 grams) and is dosed twice daily according to weight. For patients weighing less than 30 kg, administer 1 5g packet mixed into a solution per dose. In patients weighing between 30 and 65 kg, administer two 5g packets mixed into a solution per dose. And in patients weighing more than 65 kg, administer 3 5g packets mixed into a solution per dose.11The most common adverse effects (>10%) include constipation, nausea, headache, abdominal pain, cough, pain in extremities, back pain, and chest pain.11 Endari costs about $20 per 5g powder pack.9


Vosevi (sofosbuvir plus velpatasvir plus voxilaprevir)

Vosevi is a new fixed-dose combination medication that the FDA approved on July 18, 2017. Vosevi contains sofosbuvir, velpatasvir, and voxilaprevir and is indicated for the treatment of hepatits C (genotype 1, 2, 3, 4, 5, or 6) without cirrhosis or with compensated cirrhosis (Child-Pugh class A) in patients who have already tried treatment with a regimen containing an NS5A inhibitor, such as daclatasvir, elbasvir, etc., and were unsuccessful.12,13 Sofosbuvir is an inhibitor of the HCV NS5B RNA-dependent RNA polymerase, which is required for viral replication and acts as a chain terminator. Velpatasvir is an inhibitor of the HCV NS5A protein, which is also required for viral replication. Finally, voxilaprevir is an inhibitor of the NS3/4A protease, which is necessary for the proteolytic cleavage of the HCV-encoded polyprotein, which is essential for viral replication.14 Vosevi is supplied as a tablet containing 400 mg of sofosbuvir, 100 mg of velpatasvir, and 100 mg of voxilaprevir. Patients should take 1 tablet of this medication every day by mouth with food for 12 weeks.15 The most common adverse effects (>10%) include headache, fatigue, nausea, and diarrhea. Coadministration with rifampin is contraindicated when taking this medication. Other warnings include bradycardia when given with amiodarone as well as a black box warning for a risk of hepatitis B virus (HBV) reactivation. All patients should be tested for HBV prior to initiation of this treatment.15 Vosevi costs between $25,000 and $26,000 for a 28-day supply. This results in an estimated net cost of about $75,000 for a 12-week course of treatment.14

This article was written with Christopher Robinson and Charles Vawters, 2018 PharmD Candidates at the Harrison School of Pharmacy at Auburn University.


1. Brineura [prescribing information]. Novato, CA: BioMarin Pharmaceutical, Inc; 2017. Accessed October 13, 2017.

2. BioMarin. Brineura (cerliponase alfa) for CLN2 Disease. Accessed October 13, 2017.

3. Cerliponase alfa. In: Lexi-Comp Online, Lexi-Drugs [internet]. Hudson, OH: Wolters Kluwer Health/Lexi-Comp, Inc. c2017 [cited October 2, 2017].

4. Chen C. Biomarin prices orphan drug at $702,000, promises big discounts. Bloomberg. Published April 27, 2017. Accessed October 13, 2017.

5. CenterWatch. Haegarda (C1 esterase inhibitor subcutaneous [human]). Accessed October 13, 2017.

6. C1 Inhibitor (Human). In: Lexi-Comp Online, Lexi-Drugs [internet]. Hudson, OH: Wolters Kluwer Health/Lexi-Comp, Inc. c2017 [cited October 2, 2017].

7. Haegarda [prescribing information]. Marburg, Germany: CSL Behring GmbH; 2017. Accessed October 13, 2017.

8. CenterWatch. Endari (L-glutamine oral powder). Accessed October 13, 2017.

9. Glutamine. In: Lexi-Comp Online, Lexi-Drugs [internet]. Hudson, OH: Wolters Kluwer Health/Lexi-Comp, Inc. c2017 [cited October 2, 2017].

10. FDA approves new treatment for sickle cell disease [news release]. July 18, 2017. Accessed October 13, 2017.

11. Endari [prescribing information]. Torrance, CA. Emmaus Medical, Inc;. 2017. Accessed October 13, 2017.

12. FDA approves Vosevi for Hepatitis C [news release]. July 18, 2017. Accessed October 13, 2017.

13. CenterWatch Vosevi (sofosbuvir, velpatasvir, and voxilaprevir). Accessed October 13, 2017.

14. Sofosbuvir, velpatasvir, and voxilaprevir. In: Lexi-Comp Online, Lexi-Drugs [internet]. Hudson, OH: Wolters Kluwer Health/Lexi-Comp, Inc. c2017 [cited October 13, 2017].

15. Vosevi [prescribing information]. Foster City, CA. Gilead Sciences, Inc; 2017. Accessed October 13, 2017.

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