Eliglustat Tartrate

Inquiry

Cat Number

API928659705

CAS Number

928659-70-5

If you have any other questions, please contact our experts.

Product Detail Description Scientific Support Customer Q&A

CAS Number

928659-70-5

Storage

Store at room temperature

Synonyms

GENZ-112638; N0493335P3; CHEBI:83353; DTXSID50239166

Molecular Formula

C50H78N4O14

Molecular Weight

959.2

Smiles

CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O.CCCCCCCC(=O)N[C@H](CN1CCCC1)[C@@H](C2=CC3=C(C=C2)OCCO3)O.[C@@H]([C@H](C(=O)O)O)(C(=O)O)O

Appearance

White to off-white solid

Melting Point

87-92°C

Boiling Point

615.5±55.0°C (Predicted)

Relative Density

1.123±0.06 (Predicted)

General Description

Eliglustat tartrate is a small-molecule, orally active inhibitor of glucosylceramide synthase, a Golgi complex enzyme responsible for the first committed step in glycosphingolipid biosynthesis. Its structure resembles the ceramide substrate, allowing competitive inhibition.

Mechanism of Action

The drug potently and specifically binds to glucosylceramide synthase (UDP-glucose:N-acylsphingosine D-glucosyltransferase), blocking the formation of glucosylceramide from ceramide and UDP-glucose. This substrate reduction therapy decreases the synthesis of glycosphingolipids, thereby reducing the load of glucosylceramide that accumulates in the lysosomes of patients with type 1 Gaucher disease.

Application

Eliglustat tartrate is indicated for the long-term treatment of adults with type 1 Gaucher disease who are poor, intermediate, or extensive metabolizers of CYP2D6. As a substrate reduction therapy (SRT), it serves as an alternative to enzyme replacement therapy (ERT) for managing the non-neuronopathic manifestations of this lysosomal storage disorder.

In the long‑term extension phase of the ENCORE trial (up to 4 years of eliglustat treatment), 157 adults with Gaucher disease type 1 who had previously reached therapeutic goals on imiglucerase maintained stable mean hemoglobin, platelet count, spleen and liver volumes, and bone mineral density z‑scores. The composite endpoint of clinical stability was maintained in ≥85% of patients year to year. Eliglustat was well tolerated; only 4 (2.5%) withdrew due to drug‑related adverse events. No new safety concerns emerged. Eliglustat provides durable long‑term stability in this patient population.

Fig. 1 Mean for hematologic, visceral, and bone parameters over 4 years of eliglustat treatment. (Cox TM, et al., 2017)

References

Cox TM, et al. Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy. Blood. 2017;129(17):2375-2383.

Does Eliglustat Tartrate require refrigerated storage as a glucosylceramide synthase inhibitor?

No, it is stable at controlled room temperature (15-25°C). Avoid excessive heat above 30°C, which can accelerate hydrolysis of the ester groups.

Is Eliglustat Tartrate sensitive to light and moisture?

It is moderately photosensitive and slightly hygroscopic. Store in original, tightly sealed, light-resistant containers with desiccant to maintain purity.

What is the stability of Eliglustat Tartrate in capsule formulations with standard excipients?

Yes, when formulated with moisture-protective packaging.

How is the impurity eliglustat free base (loss of tartrate) monitored?

The free base is not a typical degradation product; we monitor for related process impurities using a validated HPLC method with UV detection.