Physiological Effect: Increased Coagulation Factor VIII Activity

Drugs with Physiological Effect: Increased Coagulation Factor VIII Activity

Applicant	Tradename	Generic Name	Dosage	NDA	Approval Date	TE	Type	RLD	RS	Patent No.	Patent Expiration	Product	Substance	Delist Req.	Exclusivity Expiration
Apotex Inc	DESMOPRESSIN ACETATE	desmopressin acetate	TABLET;ORAL	077414-002	Mar 7, 2006	AB	RX	No	No
Ferring Pharms Inc	DDAVP	desmopressin acetate	INJECTABLE;INJECTION	018938-001	Mar 30, 1984	AP	RX	Yes	Yes
Ferring Pharms Inc	DDAVP	desmopressin acetate	INJECTABLE;INJECTION	018938-002	Apr 25, 1995		DISCN	No	No
Ferring Pharms Inc	DDAVP	desmopressin acetate	TABLET;ORAL	019955-001	Sep 6, 1995	AB	RX	Yes	No
Ferring Pharms Inc	DDAVP	desmopressin acetate	TABLET;ORAL	019955-002	Sep 6, 1995	AB	RX	Yes	Yes
Ubi	DESMOPRESSIN ACETATE	desmopressin acetate	INJECTABLE;INJECTION	210223-001	Sep 17, 2020	AP	RX	No	No

Increased Coagulation Factor VIII Activity Market Analysis and Financial Projection

The market for drugs enhancing coagulation factor VIII (FVIII) activity is experiencing robust growth, driven by technological advancements and rising hemophilia prevalence. Here's an analysis of the market dynamics and patent landscape shaping this sector:

Market Dynamics

Growth Projections

• The global FVIII deficiency treatment market is projected to grow from USD 2.78 billion in 2025 to USD 3.94 billion by 2032 (CAGR: 5.1%), fueled by product launches and increasing hemophilia cases[1].
• The broader hemophilia treatment market is expected to reach USD 21.48 billion by 2033 (CAGR: 5.69%)[3], with the U.S. market alone valued at USD 4.21 billion in 2024 (CAGR: 6.2% through 2031)[17].

Key Drivers

1. Innovative Therapies:

   • Extended Half-Life (EHL) Products: PEGylated FVIII (e.g., Bioverativ’s Eloctate) and Fc-fusion proteins reduce infusion frequency[1][16].
   • Gene Therapies: Valoctocogene roxaparvovec (Roctavian) and efanesoctocog alfa (Altuviiio) achieved annualized bleeding rates ≤0.6[3], while SPK-8011 and SB-525 (giroctocogene fitelparvovec) show promise in early trials[16].
   • Recombinant FVIII: Dominates 50% of the U.S. market[17] due to reduced immunogenicity and improved safety.

2. Regional Trends:

   • North America holds 39.5% market share[1], driven by players like Baxter and Bayer.
   • Asia-Pacific is the second-largest region, with India reporting 100,000–125,000 hemophilia cases[1].

3. R&D Investments:

   • Global R&D spending exceeds USD 1.7 trillion[3], supporting advancements in drug discovery and clinical success rates.

Challenges

• Inhibitor Development: Up to 30% of severe hemophilia A patients develop antibodies against FVIII[5]. The EMA mandates individualized risk assessments for each product[5].
• Supply Chain Disruptions: COVID-19 impacted drug distribution, particularly in emerging markets[1].

---

Patent Landscape

Innovation Trends

1. Structural Modifications:

   • PEGylation: Enhances stability and half-life (e.g., US-20070244301A1)[11].
   • B-Domain Deletions: Truncated FVIII variants (e.g., US-5668108) retain procoagulant activity[8].
   • Mutation Engineering: Stabilizing A-domain interfaces (US-10370430-B2)[2] and glycosylation site optimization[9].

2. Production Methods:

   • Methods to boost recombinant FVIII yields 20-fold without von Willebrand factor co-expression (EPO case T0688/14)[10].

Patent Activity

• 3,424 patents were filed between 1997–2016, with 52% originating in the U.S.[12].
• Key focus areas:
  • Conjugate polymers (e.g., PEG-FVIII)[6][12].
  • Gene therapy vectors (e.g., AAV-mediated delivery)[18].

Patent Strategy	Examples
Extended Half-Life	PEGylation (US-20070244301A1), Fc-fusion proteins
Enhanced Stability	A-domain mutations (US-10370430-B2), B-domain deletions (US-5668108)
Gene Therapy	SPK-8011, BAY2599023 (AAV-FVIII)[14]

Competitive Landscape

• Major players include Novo Nordisk, Sanofi, Pfizer, and Bayer, with collaborations accelerating innovation[14].
• Litigation over recombinant DNA claims (e.g., Genetics Inst. v. Vaccines) highlights IP competitiveness[13].

---

Future Outlook

• Gene Therapies: Expected to dominate post-2030, with 75% of pipeline candidates in Phase II/III trials[14].
• Biosimilars: Patent expiries (e.g., Eloctate in 2026) may lower costs but intensify competition.

Highlight: "The continuous evolution of recombinant FVIII concentrates aims to improve efficacy, safety, and quality of life for hemophilia A patients." [4]

The convergence of advanced biologics, gene editing, and patient-centric formulations will define the next decade of FVIII therapeutics.

References

1. https://www.coherentmarketinsights.com/market-insight/rest-of-the-world-factor-viii-deficiency-treatment-market-3257
2. https://pubchem.ncbi.nlm.nih.gov/patent/US-10370430-B2
3. https://www.astuteanalytica.com/industry-report/hemophilia-treatment-market
4. https://pmc.ncbi.nlm.nih.gov/articles/PMC10497389/
5. https://www.ema.europa.eu/en/medicines/human/referrals/factor-viii
6. https://pubs.rsc.org/en/content/articlehtml/2021/na/d1na00119a
7. https://www.databridgemarketresearch.com/reports/global-von-willebrand-disease-market
8. https://patents.justia.com/patents-by-us-classification/930/100
9. https://patents.google.com/patent/US20130085110A1/en
10. https://www.epo.org/en/boards-of-appeal/decisions/t140688eu1
11. https://patents.google.com/patent/US20070244301A1/en
12. https://singep.org.br/7/wp-content/uploads/sites/3/2018/10/Singep-apresentacao-Gesiane.pdf
13. https://casetext.com/case/genetics-inst-llc-v-vaccines-1
14. https://www.prnewswire.com/news-releases/coagulation-factor-deficiency-market--us-to-have-the-lions-share-among-the-7mms--delveinsight-302193212.html
15. https://patents.justia.com/patents-by-us-classification/530/381
16. https://www.biospace.com/hemophilia-a-market-size-to-reach-usd-28-447-2-million-by-2034-impelled-by-the-inflating-demand-for-hemostatic-agents
17. https://www.globenewswire.com/news-release/2024/07/26/2919408/0/en/U-S-Hemophilia-Treatment-Market-Size-to-Worth-US-6-81-Billion-by-2031-growing-at-a-CAGR-of-6-2-CoherentMI.html
18. https://go.gale.com/ps/i.do?id=GALE%7CA572648992&sid=googleScholar&v=2.1&it=r&linkaccess=abs&issn=10870156&p=HRCA&sw=w
19. https://www.cognitivemarketresearch.com/recombinant-factor-viii-market-report