6 BioPharm International
®
Emerging Therapies eBook September 2023 www.biopharminternational.com
Industry
trends
treatment of Merkel cell carcinoma, a very rare and
aggressive form of skin cancer (7), while the two larger
companies both released treatments for certain types
of diffuse large-B cell lymphoma (DLBCL), a type of
non-Hodgkin lymphoma that is significantly more
common.
That said, epcoritamab-bysp and glofitamab-gxbm do
target different variations of DLBCL. Epcoritamab-bysp's
indication was for relapsed or refractory DLBCL not oth-
erwise specified, including DLBCL arising from indolent
lymphoma, and high-grade B-cell lymphoma after two
or more lines of systemic therapy (8). While glofitam-
ab-gxbm is also indicated for not otherwise specified
DLBCL, it's also indicated for large B-cell lymphoma
(LBCL) arising from follicular lymphoma after two or
more lines of systemic therapy (9). Of note is that both
were granted approval under the Accelerated Approval
pathway. In addition to treating a different, very rare
form of cancer, retifanlimab-dlwr received full approval
for its indication (7).
Rare diseases and other conditions
While retifanlimab-dlwr falls more clearly into the
oncological therapeutic categor y, it is not the only
rare disease t hat received approva ls. Whi le t hey
cover wildly different categories, retifanlimab-dlwr,
Pfizer's Ngenla (somatrogon-ghla), Chiesei's Lamzede
(vel m a n a se a l f a-t yc v), A s t ra zeneca's Be y for t u s
(n i rsev i m ab-a l ip), a nd Regeneron's Veopoz (po-
zelimab-bbfg) all treat rare conditions. Regeneron's
Veopoz (pozelimab-bbfg) is the first FDA-approved
treatment for CD55-deficient protein-losing enterop-
athy (PLE), also known as CHAPLE disease. The ex-
tremely painful and often life-threatening disease
af fects less than 100 patients worldwide, and po-
zelimab-bbfg received fast track, orphan drug, and
rare pediatric disease designations (10).
Neurodegenerative diseases
On Aug 24, 2023, FDA approved Tyruko (natalizum-
ab-sztn), an injection for adults with relapsing forms
of multiple sclerosis (MS). Tyruko is a biosimilar to
Tysabri (natalizumab) used in adult patients with
moderate to severe active Crohn's disease (CD) with
inf lammation symptoms who are unable to receive
normal CD therapies. Tyruko was approved to treat
three t ypes of MS: clinically isolated syndrome (a
singular, initial emergence of MS symptoms), relaps-
ing-remitting disease (episodes of new neurological
symptoms and then periods of stability), and active
secondary progressive disease (11).
In addition, Eisai's Leqembi (lecanemab-irmb),
UCB's Rystiggo (rozanolixizumab-noli), and Chiesei's
Elfabrio (pegunigalsidase alfa-iwxj) all treat neurode-
generative diseases—Alzheimer's, Generalized My-
asthenia Gravis, and Fabry Disease, respectively (1).
Looking forward
The biologic market is more active than ever, as com-
panies continue to develop new therapies both in the
United States and overseas. Amicus Therapeutics re-
ceived marketing authorization for Pombiliti (cipaglu-
cosidase alfa) (12), while Alterity Therapeutics locked in
an European Medicines Agency composition of matter
patent for their new class of iron chaperone drug candi-
dates (13). And companies continue to invest in upcom-
ing biologic treatments, such as Royalty Pharma putting
$500 million into Ferring Pharmaceutical's adstiladrin
(nadofaragene firadenovec-vncg), a gene therapy (14).
This enthusiasm for biologics development is a pos-
itive move in the market that should provide much
needed treatments for patients.
References
1. FDA. Novel Drug Approvals for 2023. FDA.gov/drugs/
new-drugs (accessed Sep. 15, 2023).
2. FDA. Novel Drug Approvals for 2022. FDA.gov/drugs/
new-drugs (accessed Sep. 15, 2023).
3. FDA. Novel Drug Approvals for 2021. FDA.gov/drugs/
new-drugs (accessed Sep. 15, 2023).
4. Playter, G. The United States and the Global Bio-
Pharma Market. BioPharm Internl. 2023, 36 (8) 32-33.
5. Sraits Research. Biologics Market. straitsresearch.
com/report/biologics-market (accessed Sep. 15, 2023).
6. BioSpace. Biologics Market Size to Hit USD 719.94
Billion by 2030. BioSpace.com, Oct. 6, 2022.
7. FDA. FDA D.I.S.C.O. Burst Edition: FDA Approval of
Zynyz (retifanlimab-dlwr) for Metastatic or Recur-
rent Locally Advanced Merkel Cell Carcinoma. Press
Release, Apr. 20, 2023.
8. FDA. FDA D.I.S.C.O. Burst Edition: FDA Approval of
Epkinly (epcoritamab-bysp) for Relapsed or Refrac-
tory Diffuse Large B-cell Lymphoma and High-grade
B-cell Lymphoma. Press Release, June 21, 2023.
9. FDA. FDA Grants Accelerated Approval to Glofitam-
ab-gxbm for Selected Relapsed or Refractory Large
B-cell Lymphomas. Press Release, June 16, 2023.
10. FDA. FDA Approves First Treatment for CD55-de-
ficient Protein-losing Enteropathy (CHAPLE dis-
ease). Press Release, Aug. 18, 2023.
11. FDA. FDA Approves Biosimilar to Treat Multiple Scle-
rosis. Press Release. Aug. 24, 2023.
12. Amicus Therapeutics. Amicus Therapeutics An-
nounces Approval and Launch of New Pompe Dis-
ease Therapy in the United Kingdom. Press Release.
Aug. 15, 2023.
13. Alterity Therapeutics. Alterity Therapeutics Granted
New Composition of Matter Patent in Europe for Neu-
rodegenerative Diseases Including Parkinson's and
Alzheimer's. Press Release. Aug. 23, 2023.
14. Playter, G. Royalty Pharma Places $500 Million Bet
on Ferring Gene Therapy. BiopharmInternational.com,
Aug. 24, 2023. ■
