New Drugs Approved by FDA in August-September 2022 with their Brand Name, Indications, Dosage, Mechanism of action- DrugsInfo

1. New Drug Approved by FDA
August 2022-September 2022
Prepared By- Akash Agnihotri

2. New Drug Approved by FDA
Olipudase alfa

3. New Drug Approved by FDA
• Drug Name: Olipudase alfa
• Brand Name: Xenpozyme™
• Approval date: 31-Aug-2022
• Indication: To treat Acid Sphingomyelinase Deficiency
• Company Name: Sanofi

4. Olipudase alfa: Detailed Indication
Olipudase alfa is a hydrolytic lysosomal
sphingomyelin-specific enzyme indicated for
treatment of non–central nervous system
manifestations of acid sphingomyelinase
deficiency (ASMD) in adult and pediatric patients

5. Acid sphingomyelinase deficiency :
info
Acid sphingomyelinase deficiency (ASMD) is a rare
progressive genetic disorder that results from a deficiency of
the enzyme acid sphingomyelinase, which is required to break
down (metabolize) a fatty substance (lipid) called
sphingomyelin.
ASMD with significant morbidity and mortality, especially
among infants and children, as many pediatric patients will not
survive to adulthood.
Signs and symptoms of ASMD may include enlarged spleen
or liver, difficulty breathing, lung infections, and unusual
bruising or bleeding, among other disease manifestations.
Current management of the disease includes palliative and
supportive care to manage the symptoms

6. Olipudase alfa
Xenpozyme® (olipudase alfa) approved by
European Commission as first and only treatment
for acid sphingomyelinase deficiency (ASMD)
Adults: Recommended starting dose:
0.1 mg/kg administered as an intravenous infusion
Pediatrics: Recommended starting dose:
0.03 mg/kg administered as an intravenous
infusion

7. Olipudase alfa: Mechanism of action
• ASMD is a lysosomal storage disease that
results from reduced activity of the enzyme
acid sphingomyelinase (ASM), caused by
pathogenic variants in the sphingomyelin
phosphodiesterase 1 gene.
• ASM degrades sphingomyelin to ceramide and
phosphocholine.

8. Olipudase alfa: Mechanism of action
• The deficiency of ASM causes an intra-
lysosomal accumulation of sphingomyelin (as
well as cholesterol and other cell membrane
lipids) in various tissues.
• Olipudase alfa provides an exogenous source of
ASM.
• Olipudase alfa is not expected to cross the
blood-brain barrier or modulate the CNS
manifestations of ASMD.

9. New Drug Approved by FDA
Spesolimab

10. New Drug Approved by FDA
• Drug Name: Spesolimab
• Brand Name: Spevigo™
• Approval date: 01-Sep-2022
• Indication: To treat generalized pustular psoriasis flares
• Company Name: Boehringer Ingelheim

11. Spesolimab: Detailed Indication
Spesolimab is an interleukin-36 receptor
antagonist indicated for the treatment of
generalized pustular psoriasis (GPP) flares in
adults

12. Generalized pustular psoriasis : Info
GPP is a rare, life-threatening neutrophilic skin disease, which is
distinct from plaque psoriasis.
It is characterized by episodes of widespread eruptions of
painful, sterile pustules (blisters of non-infectious pus).
There is a high unmet need for treatments that can rapidly and
completely resolve the signs and symptoms of GPP flares.
Flares greatly affect a person’s quality of life and can lead to
hospitalization with serious complications, including heart failure,
renal failure, sepsis, and death.

13. Spesolimab
First-in-class treatment to people living with generalized
pustular psoriasis (GPP)
Administration:
Administer as a single 900 mg dose by intravenous
infusion over 90 minutes. If flare symptoms
persist, may administer an additional intravenous
900 mg dose one week after the initial dose
Strength:
Injection: 450 mg/7.5 mL (60 mg/mL) solution in a
single-dose vial

14. Spesolimab: Mechanism of action
• Spesolimab-sbzo is a humanized monoclonal
immunoglobulin G1 antibody that inhibits
interleukin-36 (IL-36) signaling by specifically
binding to the IL36R.
• Binding of spesolimab-sbzo to IL36R prevents
the subsequent activation of IL36R by cognate
ligands (IL-36 α, β and γ) and downstream
activation of pro-inflammatory and pro-fibrotic
pathways.
• The precise mechanism linking reduced IL36R
activity and the treatment of flares of GPP is
unclear.

15. New Drug Approved by FDA
DaxibotulinumtoxinA

16. New Drug Approved by FDA
• Drug Name: Daxibotulinumtoxin-A
• Brand Name: DAXXIFY™
• Approval date: 07-Sep-2022
• Indication: To treat moderate-to-severe glabellar
lines associated with corrugator and/or procerus
muscle activity
• Company Name: Revance

17. Daxibotulinumtoxin-A
(DaxibotulinumtoxinA-lanm) for Injection
The First and Only Peptide-Formulated
Neuromodulator With Long-Lasting Results
Not recommended for use in children or
pregnant women
Dosage & Administration:
Glabellar Lines: 0.1 mL (8 Units) by intramuscular
injection into each of five sites, for a total dose
of 40 Units

18. Daxibotulinumtoxin-A- Detailed Indication
DaxibotulinumtoxinA-lanm) for injection
is an acetylcholine release inhibitor
and neuromuscular blocking agent
indicated for the temporary improvement
in the appearance of moderate to severe
glabellar lines associated with
corrugator and/or procerus muscle
activity in adult patients.

19. Glabellar lines associated with corrugator and/or
procerus muscle activity: info
The corrugator (or corrugator supercilii) muscles are a pair
of muscles located at the medial end of the eyebrow.
They have recognition as significant muscles in the
glabellar and central forehead regions.
The corrugator supercilii muscles (CSM) play a major role
in elevating the medial segment of the eyebrow,
depressing the lateral portion of the eyebrow and are
responsible for producing vertical frown lines.

20. Daxibotulinumtoxin-A: Mechanism of action
• It blocks cholinergic transmission at the neuromuscular junction
by inhibiting the release of acetylcholine.
• When injected into skeletal muscle, it is internalized into the
nerve terminal, translocates into the neuronal cytosol where it
cleaves SNAP25, a protein necessary for synaptic vesicle
membrane docking and subsequent release of acetylcholine
which produces a dose dependent decrease of muscle function.
• Recovery of activity is gradual and results from the degradation
of neurotoxin light chain in the neurons with a contribution
from the formation of axonal sprouts.
• Muscle reinnervation occurs, leading to a slow reversal of the
pharmacological effects of Daxibotuliniumtoxin-A

21. New Drug Approved by FDA
Deucravacitinib

22. New Drug Approved by FDA
• Drug Name: Deucravacitinib
• Brand Name: Sotyktu™
• Approval date: 09-Sep-2022
• Indication: To treat moderate-to-severe plaque
psoriasis
• Company Name: Bristol Myers Squibb

23. Deucravacitinib: Detailed Indication
Deucravacitinib is a tyrosine kinase 2 (TYK2)
inhibitor indicated for the treatment of adults
with moderate-to-severe plaque psoriasis who are
candidates for systemic therapy or phototherapy

24. Plaque psoriasis: Info
Psoriasis is recognized as the most prevalent immune-mediated
inflammatory disease, involving skin and joints and associated
with abnormalities of other systems.
Plaque psoriasis is the commonest clinical type of psoriasis.
Even though the condition is not life-threatening, it is difficult
to treat and response rates vary widely.
Plaque psoriasis typically presents with large oval-circular
plaques over the scalp, trunk and extensor body surface.
The plaques have an extensive amount of scaling due to the
hyperproliferation of the epidermal cells.
Most patients have acute flares and relapses of plaque
psoriasis, which may sometimes become severe and progress
into the pustular subtype.
At least 15% of patients with plaque psoriasis will develop
psoriatic arthritis.

25. Deucravacitinib
Oral Treatment for Adults with Moderate-to-
Severe Plaque Psoriasis
A first-in-class, oral, selective, allosteric
tyrosine kinase 2 (TYK2) inhibitor, is the only
approved TYK2 inhibitor worldwide and the first
innovation in oral treatment for moderate-to-
severe plaque psoriasis in nearly 10 years
Dosage & Administration:
Recommended dosage is 6 mg orally once daily,
with or without food

26. Deucravacitinib: Mechanism of action
• Deucravacitinib is an inhibitor of tyrosine kinase 2
(TYK2). TYK2 is a member of the Janus kinase (JAK)
family.
• Deucravacitinib binds to the regulatory domain of
TYK2, stabilizing an inhibitory interaction between the
regulatory and the catalytic domains of the enzyme.
• This results in allosteric inhibition of receptor-
mediated activation of TYK2 and its downstream
activation of Signal Transducers and Activators of
Transcription (STATs) as shown in cell-based assays.
• JAK kinases, including TYK2, function as pairs of homo-
or heterodimers in the JAK-STAT pathways.

27. Deucravacitinib: Mechanism of action
continues
• TYK2 pairs with JAK1 to mediate multiple
cytokine pathways and also pairs with JAK2 to
transmit signals as shown in cell-based assays.
• The precise mechanism linking inhibition of
TYK2 enzyme to therapeutic effectiveness in
the treatment of adults with moderate-to-
severe plaque psoriasis is not currently known,

28. New Drug Approved by FDA
Eflapegrastim

29. New Drug Approved by FDA
• Drug Name: Eflapegrastim
• Brand Name: Rolvedon™
• Approval date: 09-Sep-2022
• Indication: To decrease the incidence of
infection in patients with non-myeloid
malignancies receiving myelosuppressive anti-
cancer drugs associated with clinically
significant incidence of febrile neutropenia
(Chemotherapy induced neutropenia)
Company Name: Hanmi
Pharmaceutical

30. Eflapegrastim: Detailed Indication
Eflapegrastim is a leukocyte growth factor
indicated to decrease the incidence of infection,
as manifested by febrile neutropenia, in adult
patients with nonmyeloid malignancies receiving
myelosuppressive anti-cancer drugs associated
with clinically significant incidence of febrile
neutropenia

31. Chemotherapy induced neutropenia: info
Chemotherapy-induced neutropenia (CIN) is a potentially
fatal and common complication in myelosuppressive
chemotherapy.
The timing and grade of CIN may play prognostic and
predictive roles in cancer therapy.
CIN is generally characterized as a decreased absolute
neutrophil count (ANC) < 2,000 cells/mm3 in peripheral
blood. Further classification of the severity of CIN is
evaluated by the National Cancer Institute Common
Terminology Criteria for Adverse Events, version 5.0.
According to this grading system, neutropenia is classified
according to the following 4 grades:
(i) Grade 1 with an ANC of 1,500–2,000 cells/mm3, (ii)
Grade 2 with an ANC of 1,000–1,500 cells/mm3, (iii) Grade
3 with an ANC of 500–1,000 cells/mm3, and (iv) Grade 4
with an ANC < 500 cells/mm3.

32. Eflapegrastim
First novel Long-Acting GCSF (LA-GCSF) product
approved in over 20 years
Recommended Dose:
13.2 mg administered subcutaneously once per
chemotherapy cycle
Dosage:
Injection: 13.2 mg/0.6 mL solution in a single-dose
prefilled syringe

33. Eflapegrastim : Mechanism of action
Eflapegrastim-xnst is a recombinant human
granulocyte growth factor that binds to G-CSF
receptors on myeloid progenitor cells and neutrophils,
triggering signaling pathways that control cell
differentiation, proliferation, migration and survival

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