1. The document presents an overview of 14 newer drugs approved by the US FDA in 2016 for various therapeutic indications. It provides details on the drug name, approval date, classification, indication, mechanism of action, adverse effects and dosing for each drug. The drugs are for treating conditions like hepatitis C, epilepsy, anthrax infection, psoriasis, asthma, and various types of cancer. 2. It also includes two diagnostic imaging agents - one for detecting recurrent prostate cancer and another for neuroendocrine tumors. The last drug discussed is an ophthalmic solution for treating dry eye disease. In summary, the document outlines the key details of 14 novel pharmaceutical agents approved in the US in 2016 for a range of

1. NEWER DRUGS APPROVED
BY US-FDA: A PRECISE
OUTLOOK(PART-1)
PRESENTED BY:
VISHNU.R.NAIR,
4TH YEAR PHARM.D,
NATIONAL COLLEGE OF PHARMACY(NCP)

2.  GENERAL INTRODUCTION : 
▪ INNOVATION drives PROGRESS
▪ As far as INNOVATION regarding DEVELOPMENT of NEW DRUGS and THERAPEUTIC
BIOLOGICAL PRODUCTS is concerned , FDA’s CENTER FOR DRUG EVALUATION and
RESEARCH (CDER) supports the pharmaceutical industry at every step of the process
▪ With its understanding of the science used to create new products, testing &
manufacturing procedures, & the diseases and conditions that new products are designed
to treat, FDA provides SCIENTIFIC & REGULATORY ADVICE needed to bring new
therapies to market.
▪ Thus, I am going to present the NEWER DRUGS APPROVED BY US-FDA for the year
2016…………………………………………….

3. US-FDA APPROVED DRUGS
IN
2016……………………………………

4. 1. ELBASVIR+GRAZOPREVIR(ZEPATIER):
- ELBASVIRCLASS : NS5A Inhibitor (Non-structural protein 5A)
- GRAZOPREVIRCLASS: NS3-4A inhibitor (Serine protease)
- APPROVEDON : 28.01.2O16
- INDICATION : To treat patients with CHRONIC HEPATITIS C VIRUS (HCV)
- Effective against HCV GENOTYPES 1 & 4
- Used in adults
- MOA :
Elbasvir  targets NS5A  Prevents TRANSCRIPTION of HCV RNA  Prevents
VIRION ASSEMBLY

5. - GRAZOPREVIR  Blocks NS3-4A protease  prevents CLEAVAGE OF
POLYPROTEINS involved in HCV REPLICATION
- ADRs : Headache, tiredness
- INTERACTION : Drug + RIBAVIRIN  LOW RBC COUNT
- DOSE : 50 mg (ELBASVIR) + 100 mg (GRAZOPREVIR) ; OD ; For 12
weeks……………..

6. 2. BRIVIACT (BRIVARACETAM) :
- CLASS : RACETAM DERIVATIVE
- It’s a chemical analogue of LEVETIRACETAM
- INDICATION : To treat PARTIAL ONSET SEIZURES in patients aged 16 YEARS
OR OLDER with EPILEPSY
- APPROVED ON : 18.02.2016
- MOA :
DRUG  Binds to SYNAPTIC VESICLE GLYCOPROTEIN 2A (SV2A)  Reduces
EXCITATORY NEUROTRANSMITTER ACTIVITY , along with enhancing
SYNAPTIC DEPRESSION, during high frequency discharges that occur during epilepsy
- Drug is 20 times more efficient than LEVETIRACETAM
- ADRs : Sleepiness, dizziness, co-ordination problems

7. - INTERACTIONS:
a. DRUG + CARBAMAZEPINE  Increased EXPOSURE to ACTIVE
METABOLITE OF CBZ (CBZ-EPOXIDE)  Reduces its TOLERABILITY
b. DRUG + PHENYTOIN  Increases PHENYTOIN levels
- Maximum dose : 100 mg BID/ day(PO)
- If TOLERABILITY is poor, dose can be tapered to 25 mg BID/day
- Initial dose : 50 mg BID/ day…………………….

8. 3. OBLITOXAXIMAB (ANTHIM) :
- APPROVED ON : 18/2/2016
- INDICATION : To TREAT INHALATIONALANTHRAX in COMBINATION with
APPROPRIATE ANTIBACTERIAL DRUGS
- CLASS : MONOCLONALANTIBODY ANTHRAX ANTITOXIN
- Effective against BACILLUS ANTHRACIS
- MOA :
Drug  binds to PROTECTIVE ANTIGEN(PA) COMPONENT of ANTHRAX TOXIN
 Neutralizes toxin  prevents toxin’s entry into susceptible cells  avoids its
proliferation  prevents TISSUE DAMAGE
- ADR : Hypersensitivity, anaphylaxis
- DOSE : 16 mg/kg i.v over 90 mins………………………

9. 4. IXEKIZUMAB(TALTZ):
- APPROVED ON : 22.03.2016
- INDICATION : To treat adults with MODERATE-SEVERE PLAQUE PSORIASIS
- CLASS : INTERLEUKIN-17 INHIBITOR; HUMANIZED MONOCLONAL
ANTIBODY
- MOA :
a. IL-17  Cytokine, that stimulates PROLIFERATION & ACTIVATION of
KERATINOCYTES in skin
b. Drug  binds to IL-17  blocks its action
c. Action is similar to that of BRODALUMAB
- ADR : Increased rate of infections, injection site pain, oropharyngeal pain
- INTERACTION : Drug + WARFARIN  Reduced WARFARIN levels

10. - DOSE : 160 mg s.c (in the form of two 80 mg injections)  then reduce to 80 mg s.c
5. RESLIZUMAB(CINQAIR):
- APPROVED ON : 23/3/2016
- CLASS : INTERLEUKIN-5 INHIBITOR; HUMANIZED MONOCLONAL
ANTIBODY
- INDICATION: To treat SEVERE EOSINOPHILIC ASTHMA
- MOA:
a. IL-5 is a major cytokine, responsible for GROWTH, DIFFERENTIATION,
RECRUITMENT, ACTIVATION & SURVIVAL of EOSINOPHILS  Causes
increased EOSINOPHIL levels  increases AIRWAY INFLAMMATION
b. Drug  binds to IL-5  Blocks it  reduces EOSINOPHILIC LEVELS

11. - ADRs:
a. Oropharyngeal pain
b. Muscle fatigue
c. Neck pain
- DOSE : 3 mg/kg i.v every 4 weeks (infuse for 20-50 mins)……
6. DEFIBROTIDE SODIUM (DEFITELIO):
- APPROVED ON : 30.03.2016
- INDICATION : To treat adults & children, who develop HEPATIC VENO-
OCCLUSIVE DISEASE, with KIDNEY/ LUNG ABNORMALITIES , on receiving
HEMATOPOEITIC STEM CELL TRANSPLANTATION (Stem cell transplant, from
blood/ bone marrow)

12. - HEPATIC VENO-OCCLUSIVE DISEASE is also known as SOS (SINUSOIDAL
OBSTRUCTION SYNDROME)
- Fatal form of hepatic injury, associated with ABDOMINAL PAIN, PORTAL
HYPERTENSION, and INCREASE IN SERUM ENZYME LEVELS
- DRUG CLASS : ANTICOAGULANT
- Derived from COW LUNG/ PORCINE MUCOSA
- MOA:
Drug has 4 actions:
a. Drug  increases levels of PGI2 Relaxes smooth muscles of blood vessels 
prevents PLATELET ADHERENCE
b. Drug  increases levels of PGE2  Inhibits PLATELET AGGREGATION
c. Drug  increases levels of PROSTACYCLIN  Inhibits platelet aggregation
d. Drug  increases function of t-PA

13. - ADRs:
a. Increased bleeding
b. Increased bruising tendency
c. Low B.P
- DOSE: 6.25 mg/kg i.v every 6 hours, infused for 2 hours………………..
7. VENETOCLAX(VENCLEXTA):
- APPROVED ON: 11.04.2016
- INDICATION : For CHRONIC LYMPHOCYTIC LEUKEMIA patients, with a
SPECIFIC CHROMOSOME ABNORMALITY
- CLASS : BCL-2 INHIBITOR

14. - MOA:
a. In patients with CHROMOSOME ‘17p’ DELETION(MUTATION)  Increases
levels of ANTI-APOPTOPIC B-CELL LYMPHOMA-2 (BCL-2) PROTEIN 
Causes CLL
b. Drugs  blocks BCL-2  Causes PROGRAMMED CELL DEATH of CLL CELLS
- ADRs:
a. NEUTROPENIA
b. URTI
c. THROMBOCYTOPENIA
d. FERTILITY PROBLEMS IN MALES
e. TUMOR LYSIS SYNDROME (Metabolic abnormality, that occurs as a complication
of CANCER CHEMOTHERAPY, where large amounts of TUMOR CELLS are
KILLED OFF(LYSED), simultaneously by the treatment, causing release of their
contents into BLOODSTREAM………………….)

15. - DOSE :
a. For 1st week : 20 mg PO OD
b. For 2nd week : 50 mg PO OD
c. For 3rd week : 100 mg PO OD
d. For 4th week : 200 mg PO OD
e. For 5th week : 400 mg PO OD…………………………..

16. 8. PIMAVANSERIN(NUPLAZID):
- APPROVED ON : 29.04.2016
- INDICATION : To treat HALLUCINATIONS & DELUSION associated with
PSYCHOSIS, experienced by people with PARKINSON’S DISEASE
- CLASS : NON-DOPAMINERGIC ATYPICAL ANTI-PSYCHOTIC
- MOA:
a. Drug  behaves as SELECTIVE INVERSE AGONIST & ANTAGONIST of 5-
HT(2A) receptor  treats HALLUCINATIONS and DELUSIONS associated with
PD
- ADRs : PERIPHERAL EDEMA, CONSTIPATION
- DOSE : 34 mg PO every day…………………………

17. 9. ATEZOLIZUMAB (TECENTRIQ):
- APPROVED ON : 18.05.2016
- INDICATION : To treat UROTHELIAL CARCINOMA(Most common type of
BLADDER CANCER)
- CLASS : IgG1 ISOTYPE HUMANIZED MONOCLONALANTIBODY
- MOA:
a. PD-L1(Programmed cell death ligand-1)  highly expressed in some tumors 
reduces activation of cytotoxic T-cells(that can recognize and attack the cancer)
b. Drug  BLOCKS INTERACTION of PD-L1 with PROGRAMMED CELL DEATH
PROTEIN-1 (PD-1) & CD-80  Removes INHIBITORY EFFECT of PD-L1 
Causes ANTI-TUMOR response…………………………….
- ADRs: Anorexia, URI
- DOSE : 1,200 mg i.v every 3 weeks (as 60 mins. Infusion)…………………………

18. 10. DACLIZUMAB (ZINBRYTA):
- CLASS : MONOCLONALANTIBODY
- APPROVED ON : 27.05.2016
- INDICATION : To treat MULTIPLE SCLEROSIS & its RELAPSE
- MOA:
a. Drug  binds to IL-2 receptor SUBUNIT (CD-25)  Blocks its activity
b. CD-25  Expressed in high levels in T-CELLS , which become abnormally activated
in MULTIPLE SCLEROSIS
- MULTIPLE SCLEROSIS is a DEMYELINATING DISEASE, in which INSULATING
COVERS of NEURONS in BRAIN & SPINAL CORD are DAMAGED  Disrupts
ability of parts of NERVOUS SYSTEM to inter-communicate  results in PHYSICAL,
MENTAL, PSYCHIATRIC PROBLEMS, along with DOUBLE VISION , BLINDNESS
in 1 eye, etc

19. - ADRs:
a. NASOPHARYNGITIS (28%)
b. URTI (9-17%)
c. BRONCHITIS (7%)
- DOSE : 150 mg s.c ONCE MONTHLY………………………

20. 11. OBETICHOLIC ACID (OCALIVA):
- CLASS : FARNESOID ‘X’ RECEPTOR AGONIST; GASTROINTESTINAL AGENT
- APPROVED ON : 27.05.2016
- INDICATION : To treat CHRONIC LIVER DISEASE
- MOA:
a. FXR is a NUCLEAR RECEPTOR expressed in the LIVER, INTESTINE, KIDNEY
& ADIPOSE TISSUE , that regulates TARGET GENES , responsible for the
CONTROL OF BILE ACID SYNTHESIS , its TRANSPORT, LIPID METABOLISM
& GLUCOSE HOMEOSTASIS
b. Drug  increases FXR activation  SUPPRESSES SYNTHESIS of BILE ACIDS in
HEPATOCYTES
c. Drug  also increases TRANSPORT OF BILE ACIDS out of HEPATOCYTES 
Reduces exposure of the HEPATOCYTES to BILE ACID……………………….

21. - ADRs:
a. PRURITUS (56-70%)
b. FATIGUE (19-25%)
c. REDUCED HDL-CHOLESTEROL (9-20%)
- DOSE : 5 mg PO every day……………………….

22. 12. FLUCICLOVINE F-18 (AXUMIN):
- APPROVED ON : 27.05.2016
- INDICATION : A new DIAGNOSTIC IMAGING AGENT, to detect RECURRENT
PROSTATE CANCER
- CLASS : DIAGNOSTIC IMAGING AGENTS
- MOA:
a. SYNTHETIC AMINO ACID
b. It is labelled with RADIO-ISOTOPE F-18 for PET-IMAGING
c. Agent  taken up by PROSTATE CANCER CELLS , using TRANSPORTERS
(LAT-1, ASCT-2)  Helps in determining the levels of the TRANSPORTERS,
compared to surrounding normal tissues………………………
- ADRs : Injection site pain, erythema

23. - DOSE : 370 mBq i.v BOLUS…………………….
13. GALLIUM GA-68 DOTETATE (NETSPOT):
- APPROVED ON : 01.06.2016
- INDICATION : DIAGNOSTIC IMAGING AGENT, to detect RARE
NEUROENDOCRINE TUMORS
- MOA:
a. Agent  binds with HIGH AFFINITY to Cells expressing SOMATOSTATIN
RECEPTORS SUBTYPE 2  helps diagnose MALIGNANT CELLS(Which
overexpress SSTR-2)
b. Allows PET imaging
- DOSE : 200 mBq , as i.v bolus injection………………………..

24. 14. SOFOSBUVIR & VELPATASVIR
(EPCLUSA):
- APPROVED ON : 28.06.2016
- INDICATION : To treat all 6 MAJOR FORMS OF HCV
- CLASS : HCV POLYMERASE INHIBITORS; HCV-NS5A INHIBITORS
- MOA:
a. VELPATASVIR  Blocks NS5A  suppresses HCV replication
b. SOFOSBUVIR  Blocks HCV NS5B RNA- dependent POLYMERASE 
suppresses VIRAL REPLICATION
- ADRs:
a. Anemia (26%)
b. Headache (22%)

25. - DOSE :
a. FOR PATIENTS , WITH CIRRHOSIS : DRUG (400 MG SOFOSBUVIR + 100 mg
VELPATASVIR) + RIBAVIRIN, with FOOD, for 12 weeks
b. FOR PATIENTS, WITHOUT CIRRHOSIS : Same dose of drug(excluding
RIBAVIRIN) , for 12 weeks……………….
15. LIFITEGRAST OPHTHALMIC SOLUTION
(XLIDRA):
- APPROVED ON : 11.07.2016
- INDICATION : To treat signs and symptoms of DRY EYE DISEASE
- DRY EYE DISEASE :
a. It’s a condition, that involves DEFICIENCY OF TEARS

26. b. Main symptom usually involves SCRATCHY / SANDY feeling , as if something is in the
eye
c. Other symptoms include:
• Stinging or burning of eye
• Episodes of EXCESS TEARING, followed by periods of VERY DRY SENSATION
- DRUG CLASS : LFA-1 blocker; OPHTHALMIC AGENT
- MOA :
a. LFA-1 (Lymphocyte function associated antigen-1) is a cell surface protein found in
leukocytes
b. LFA-1  Binds to ICAM-1(Intercellular adhesion molecule-1)  caused adhesion of
ICAM-1 to T-CELLS Causes T-CELACTIVATION  Causes secretion of
INFLAMMATORY CYTOKINES Migration to TARGET TISSUES  Causes
TISSUE DAMAGE

27. c. Drug  blocks the INTERACTION of LFA-1 with ICAM-1  Prevents T-CELL
ACTIVATION  IMMUNOMODULATORY ACTION
- ADRs:
a. Instillation site reactions (5-25%)
b. Dysgeusia (5-25%)
c. Reduced visual acuity (5-25%)
- DOSE: Instill 1 gtt (guttae) every 12 hours, into each eye………………..

28. 16. LIXISENATIDE (ADLYXIN):
- APPROVED ON : 27.07.2016
- INDICATION : To improve BLOOD SUGAR LEVELS (GLYCEMIC CONTROL)
- CLASS : GLUCAGON-LIKE PEPTIDE-1 AGONIST; ANTI-DIABETIC
- MOA:
a. Drug  agonized GLP-I receptor  increases GLUCOSE-DEPENDENT INSULIN
RELEASE
b. Drug  also reduces GLUCAGON secretion
c. Drug  slows GASTRIC EMPTYING
- ADRs:
a. NAUSEA (25%)

29. b. HYPOGLYCEMIA:
• If given with INSULIN +/- SULFONYLUREA  47.2%
• If given with INSULIN + /-METFORMIN  28.3%
• If given with INSULIN GLARGINE +/- METFORMIN +/- THIAZOLIDINEDIONE
(22%)
• If given with SULFONYLUREA +/- METFORMIN  14.5%
c. Constipation (2%)
- DOSE :
a. STARTING DOSE : 10 mcg s.c every day for first 14 days  increase dose to 20 mg
s.c every day(from the FIFTEENTH DAY)……………………………

30. 17. ETEPLIRSEN (EXONDYS 51):
- APPROVED ON : 19.09.2016
- INDICATION : To treat patients with DUCHENNE MUSCLE DYSTROPHY
- DUCHENNE MUSCULAR DYSTROPHY(DMD):
a. Best known form of MUSCLE DYSTROPHY
b. Occurs due to a MUTATION in a gene on the X-CHROMOSOME  Prevents
production of DYSTROPHIN (A normal protein in muscle)
c. DMD affects boys, and very rarely , girls
d. Typically appears at age 2
e. Symptoms include WEAKNESS IN PELVIS & UPPER LIMBS, CLUMSINESS,
FREQUENT FALLING, GENERALIZED WEAKNESS, etc
f. Some patients may have MILD MENTAL RETARDATION

31. g. Most patients with DMD die in their early twenties, due to MUSCLE-RELATED
BREATHING & HEART PROBLEMS
h. As DMD progresses  a wheelchair may be needed
i. There is no cure for DMD
j. Current treatment is directed towards symptoms, like assisting with MOBILITY,
PREVENTING SCOLIOSIS, and providing PULMONARY
THERAPY(RESPIRATORY TOILET)
- DRUG CLASS : MORPHOLINO ANTISENSE OLIGOMERS; NEUROLOGICAL
AGENTS
- MOA:
a. Drug  binds to EXON-51 of DYSTROPHIN PRE- MRNA  Causes EXON
SKIPPING during MRNA PROCESSING  Allows production of INTERNALLY
TRUNCATED DYSTROPHIN PROTEIN

32. b. EXON SKIPPING : Form of RNA SPLICING, that causes cells to SKIP OVER
FAULTY SECTIONS of GENETIC CODE, leads to a TRUNCATED, FUNCTIONAL
PROTEIN, despite the GENETIC MUTATION
c. TRUNCATED PROTEIN: A protein, SHORTENED BY MUTATION, which
specifically induces PREMATURE TERMINATION OF MESSENGER RNA
TRANSLATION
- ADRs:
a. BALANCE DISORDER (38%)
b. CONTACT DERMATITIS (25%)
c. Elevated temperature(on days of infusion)
- DOSE : 30 mg/kg i.v once weekly……………………………………….

33. 18. OLARATUMAB (LARTRUVO):
- APPROVED ON : 19.10.2016
- INDICATION : To treat adults with certain types of SOFT TISSUE SARCOMA
- CLASS : MONOCLONALANTIBODY; ANTINEOPLASTICS
- MOA:
a. PLATELET DERIVED GROWTH FACTOR RECEPTOR-ALPHA(PDGFR-
ALPHA) is a TYROSINE KINASE RECEPTOR expressed in MESENCHYMAL
CELLS
b. PDGFR- ALPHA signaling  plays role in CELL GROWTH, CHEMOTAXIS, &
MESENCHYMAL STEM CELL DIFFERENTIATION
c. PDGFR-ALPHA has been detected on some tumor cells (including sarcomas)  here,
SIGNALLING contributes to CANCER CELL PROLIFERTION, METASTASIS, and
MAINTENANCE of TUMOR MICROENVIRONMENT

34. d. Drug is a HUMAN IgG1 monoclonal antibody  binds SPECIFICALLY TO
PDGFR-ALPHA  Prevents its binding of the RECEPTOR by PDGF-AA, BB , CC
ligands  prevents its activation  prevents its signaling pathways
- ADRs:
a. LYMPHOPENIA (77%)
b. NAUSEA (73%)
c. ALOPECIA (52%)
d. NEUTROPENIA (65%)
e. MUSCULOSKELETAL PAIN (64%)
- DOSE :
15 mg/kg i.v , infused over 1 hour on DAYS ‘1’ & ‘8’ of each 21 day
cycle……………………….

35. 19. BEZLOTOXUMAB (ZINPLAVA):
- APPROVED ON : 21.10.2016
- INDICATION : To reduce the incidence of CLOSTRIDIUM DIFFICILE infection, in
patients, aged 18 years or older
- CLASS : MONOCLONALANTIBODY
- MOA:
a. Human monoclonal antibody
b. Drug  binds to CLOSTRIDIUM DIFFICILE TOXIN ‘B’  Neutralizes its effects
c. Drug  does not bind to toxin ‘A’
- ADRs:
a. HEART FAILURE (12.7%)
b. PYREXIA (5%)

36. - DOSE:
a. Indicated to reduce RECURRENCE OF CDI in adults, who are receiving
antibacterial drug treatment for CDI, & are at high risk of RECURRENCE
b. 10 mg/kg I.V over 1 hour, as a single dose………………………

37.  BIBLIOGRAPHY/ REFERENCE  :
▪ www.fda.gov/Drugs/DevelopmentApproval Process/DrugInnovation/ucm483775.htm
▪ www.emedicine.net
▪ www.healthline.com
▪ www.drugs.com
▪ www.drugbank.com
▪ www.MEDGENERA.com

38. THANK YOU !!!  