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Special Report Special Report

genetic disorder that prevents the (crinecerfont) for treating classic candidates are projected to make up
body from breaking down fats; congenital adrenal hyperplasia, a 20% of global pipeline forecast sales
 Ispen’s Iqirvo (elafi branor) for group of hereditary genetic dis- over the rest of this decade – in line
treating primary biliary cholangitis orders affecting the adrenal glands; with the category’s share of 2030 pre-
(a rare liver disease) in combination  Servier’s Voranigo (vorasidenib) scription pharmaceutical sales, accord-
with ursodeoxycholic acid; for treating Grade 2 astrocytoma ing to the Evaluate report.
 ITF Therapeutics’ (Italfarmaco) or oligodendroglioma, two types of
Duvyzat (givinostat) for treating low-grade brain tumors; However, as the Evaluate report
Duchenne muscular dystrophy, a  Syndax Pharmaceuticals’ Revuforj points out, there are certain policy un-
severe, inherited disease that causes (revumenib) for treating relapsed or certainties in the US that may affect
progressive muscle weakness and refractory acute leukemia; forecasts for bio/pharmaceuticals over-
degeneration;  Vertex Pharmaceuticals’ Alyftrek all and orphan drugs specifi cally. These
 Jazz Pharmaceuticals’ Ziihera (vanzacaftor, tezacaftor, and deuti- relate to staffi ng and funding levels at
(zanidatamab-hrii) for treating vacaftor) for treating cystic fi brosis; the US Food and Drug Administration
previously treated, unresectable or  X4 Pharmaceuticals’ Xolremdi to facilitate product development and
metastatic HER2-positive (IHC 3+) (mavorixafor) for treating WHIM reviews, US government funding
Source: Centre for Drug Evaluation and Research, US Food and Drug Administration biliary tract cancer, a rare and ag- syndrome (warts, hypogammaglobu- under the National Institutes of Health
gressive type of cancer that affects linemia (low levels of immuno- and other programs that support early
Fig. 3: Large Bio/Pharma companies: New Ophan Drug Approvals in 2024* the organs of the biliary system, globulins), infections, and myelo- drug research and development, and
companies, and 18, or 69%, were from the body’s immune system attacks for treating esophageal squamous which includes the gallbladder and kathexi (a reduction of circulating potential changes to drug pricing mea-
mid-to-smaller companies. the small bile ducts in the liver; cell carcinoma; bile ducts; white blood cells);and sures under the Infl ation Reduction Act
 Merck & Co.’s Winrevair (sotater-  BridgeBio Pharma’s Attruby (aco-  Merus’ Bizengri (zenocutuzumab-  Zerva Therapeutics’ Miplyffa (ari- (IRA), which included the creation of
Eight large bio/pharma compa- cept-csrk) for treating pulmonary ramidis) for treating cardiomyo- zbco) for treating certain forms of moclomol), for treating Niemann- the Medicare Prescription Drug Nego-
nies – Amgen, Astellas, AstraZeneca, arterial hypertension; pathy of wild-type or variant trans- unresectable or metastatic non- Pick disease type C, a rare neurode- tiation Program, which authorized the
Gilead Sciences, Merck & Co., Novo  Novo Nordisk’s Alhemo (conci- thyretin-mediated amyloidosis, a small cell lung cancer and pancreatic generative disorder. US government to negotiate prices for
Nordisk, Pfi zer, and Roche – each had zumab-mtci) for preventing or re- progressive heart condition where cancer in patients with a rare gene- certain prescription drugs under Medi-
one new orphan drug approval in 2024 ducing the frequency of bleeding misfolded transthyretin proteins tic alteration, NRG1 fusion; A look ahead care, the US federal health insurance
(see Figure 3). episodes in patients with hemophilia accumulate as amyloid deposits in  Neurocrine Biosciences’ Crenessity On a pipeline basis, orphan drug program for people 65 or older.
A or B; the heart muscle;
New drug approvals from the large  Pfi zer’s Hympavzi (marstacimab-  Day One Biopharmaceuticals’
bio/pharmaceutical companies in 2024 hncq) also for preventing or reduc- Ojemda (tovorafenib) for treating Chemical Weekly | Import-Export Data
were: ing bleeding episodes related to relapsed or refractory pediatric low-
 Amgen’s Imdelltra (tarlatamabdlle) hemophilia A or B; grade glioma (i.e., a brain tumor);
for treating extensive stage  Roche’s Piasky (crovalimab-akkz)  Geron’s Rytelo (imetelstat) for Market Surveys | Directories
small-cell lung cancer; for treating paroxysmal nocturnal treating low to intermediate-1
 Astellas’s Vyloy (zolbetuximab- hemoglobinuria, a rare blood dis- risk myelodysplastic syndromes, a Business Forums | Expositions
clzb) in combination with fl uoro- ease that causes red blood cells to group of progressive bone marrow
pyrimidine and platinum-containing break down. disorders;
chemotherapy as a fi rst-line treat-  Incyte’s Niktimvo (axatilimab-csfr) The only organisation in India catering exclusively to the needs of the entire chemical industry
ment of adults with locally ad- Eighteen mid-to-small bio/pharma- for treating chronic graft-versus-
vanced unresectable or metastatic ceutical companies had a new host disease after failure of at least Contact:
HER2-negative gastric or gastro- orphan drug approval in 2024. The two prior lines of systemic therapy;
esophageal junction adenocar- companies and products are outlined  IntraBio’s Aqneursa (levacetylleu- SEVAK PUBLICATIONS PVT. LTD.
cinoma; below: cine) for treating Niemann-Pick dis-
 AstraZeneca’s Voydeya (danicopan)  Ascendis Pharma’s Yorvipath (palo- ease type C, a rare genetic disease 602-B, Godrej Coliseum, K.J. Somaiya Hospital Road,
for treating extravascular hemolysis pegteriparatide) for treating hypo- resulting in progressive neurologi- Behind Everard Nagar, Sion (E),
with paroxysmal nocturnal hemo- parathyroidism, a rare disease cal symptoms and organ dysfunc- Mumbai 400 022.
globinuria, a rare blood disease; where the parathyroid glands tion;
 Gilead Sciences’ Livdelzi (sela- behind the thyroid do not produce  Ionis Pharmaceuticals’Tryngolza Phone: +91-22-24044471 / 72
delpar) for treating primary biliary enough parathyroid hormone; (olezarsen) for treating familial Email: admin@chemicalweekly.com
cholangitis, a liver disease where  BeOne’s Tevimbra (tislelizumabjsgr) chylomicronemia syndrome, a rare

164 Chemical Weekly May 27, 2025 Chemical Weekly May 27, 2025 165

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