N-acetylgalactosamine 4-sulfatase — Competitive Intelligence Brief

N-acetylgalactosamine 4-sulfatase (N-acetylgalactosamine 4-sulfatase) competitive landscape: 6 comparators, recent regulatory actions, upcoming PDUFA, patent timeline. Class: Enzyme replacement therapy. Area: Rare genetic disease / Lysosomal storage disorder.

phase 3 Enzyme replacement therapy GALNS (N-acetylgalactosamine 4-sulfatase) Rare genetic disease / Lysosomal storage disorder Small molecule

Target snapshot

N-acetylgalactosamine 4-sulfatase (N-acetylgalactosamine 4-sulfatase) — BioMarin Pharmaceutical. N-acetylgalactosamine 4-sulfatase is an enzyme replacement therapy that replaces the deficient GALNS enzyme to break down keratan sulfate and chondroitin 6-sulfate in lysosomal storage disease.

Comparator set (6 drugs)

Drug	Generic	Sponsor	Phase	Class	Target	Approval / PDUFA
N-acetylgalactosamine 4-sulfatase TARGET	N-acetylgalactosamine 4-sulfatase	BioMarin Pharmaceutical	phase 3	Enzyme replacement therapy	GALNS (N-acetylgalactosamine 4-sulfatase)
ALGLUCOSIDASE ALFA (MYOZYME)	ALGLUCOSIDASE ALFA (MYOZYME)	Genzyme, a Sanofi Company	marketed	Enzyme replacement therapy	Acid alpha-glucosidase (GAA)
Myozyme® (alglucosidase alfa)	Myozyme® (alglucosidase alfa)	Genzyme, a Sanofi Company	marketed	Enzyme replacement therapy	Acid alpha-glucosidase (GAA); lysosomal glycogen
Elelyso	Taliglucerase Alfa	Pfizer Inc.	marketed	Enzyme replacement therapy	Glucocerebroside (substrate); mannose receptors on cell surface
Fabrazyme (agalsidase beta)	Fabrazyme (agalsidase beta)	Genzyme, a Sanofi Company	marketed	Enzyme replacement therapy (ERT); recombinant enzyme	α-galactosidase A (GLA gene product); substrate: globotriaosylceramide (GL-3)
alpha-1-Proteinase Inhibitor	alpha-1-Proteinase Inhibitor	Institute for Human Genetics and Biochemistry	marketed	Protease inhibitor / Enzyme replacement therapy	Neutrophil elastase
RDD to Palynziq	RDD to Palynziq	BioMarin Pharmaceutical	marketed	Enzyme replacement therapy	Phenylalanine ammonia-lyase (PAL)

Recent regulatory actions (last 90 days)

No regulatory actions in the last 90 days for this set.

Upcoming PDUFA dates (next 180 days)

No PDUFA dates in the next 180 days for this set.

Patent timeline (forward window)

No upcoming patent expiries for this set.

Sponsor landscape (Enzyme replacement therapy class)

1. BioMarin Pharmaceutical · 5 drugs in this class
2. Sanofi · 3 drugs in this class
3. JCR Pharmaceuticals Co., Ltd. · 2 drugs in this class
4. Genzyme, a Sanofi Company · 2 drugs in this class
5. Pfizer Inc. · 1 drug in this class
6. Shire · 1 drug in this class
7. Alexion Pharmaceuticals, Inc. · 1 drug in this class
8. University of Missouri-Columbia · 1 drug in this class
9. Eurofarma Laboratorios S.A. · 1 drug in this class
10. ISU Abxis Co., Ltd. · 1 drug in this class