Amicus Therapeutics, Inc.

Kamlesh A Sheth

Philip J Rybczynski

Robert E Boyd

Benjamin Mugrage

Xiaoxiang Zhu

Gary Lee

David Palling

James Cartwright

Wendy Cross

Clive King

Jian-Qiang Fan

Sergey Tesler

Alexander Tretyakov

Dan Fuerst

Sergey Tesier

Benjamin Mugrace

Provided are methods of producing Active Pharmaceutical Ingredient (API) grade migalastat hydrochloride, and for purifying intermediate grade migalastat hydrochloride. Further provided are methods of producing [(2R,3S,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol hydrochloride (lucerastat hydrochloride) and other 1-deoxygalactonojirimycin compounds, as well as methods of purifying intermediate grade lucerastat hydrochloride and other 1-deoxygalactonojirimycin compounds.

Highly purified batches of pharmaceutical grade 1-deoxygalactonojirimycin compounds

Provided are methods of producing a batch of 1,2,3,6-tetrapivaloyl-D-galactofuranoside; 5-azido-5-deoxy-1,2,3,6-tetrapivaloyl-D-galactofuranoside; intermediate grade migalastat hydrochloride; and/or migalastat hydrochloride. Also provided are methods of determining the purity of a batch of 1,2,3,6-tetrapivaloyl-D-galactofuranoside; 5-azido-5-deoxy-1,2,3,6-tetrapivaloyl-D-galactofuranoside; intermediate grade migalastat hydrochloride; and/or migalastat hydrochloride. Also provided are methods of distributing a batch of 1,2,3,6-tetrapivaloyl-D-galactofuranoside; 5-azido-5-deoxy-1,2,3,6-tetrapivaloyl-D-galactofuranoside; intermediate grade migalastat hydrochloride; and/or migalastat hydrochloride. Also provided are methods of assessing suitability of migalastat hydrochloride for medical use.

Highly purified batches of pharmaceutical grade migalastat and methods of producing the same

The present invention provides a method for increasing the activity of a mutant or wild-type &#x3b1;-glucosidase enzyme in vitro and in vivo by contacting the enzyme with a specific pharmacological chaperone which is a derivative of 1-deoxynojirimycin. The invention also provides a method for the treatment of Pompe disease by administration of chaperone small molecule compound which is a derivative of 1-deoxynojirimycin. The 1-deoxynojirimycin derivative is substituted at the N or C1 position. Combination therapy with replacement &#x3b1;-glucosidase gene or enzyme is also provided.

Method for the treatment of Pompe disease using 1-deoxynojirimycin derivatives

Described are novel salts of the compound (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease and/or Parkinson's disease.

Compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system

A method for preparing isofagomine, its derivatives, intermediates and salts thereof using novel processes to make isofagomine from D-(&#x2212;)-arabinose and L-(&#x2212;)-xylose.

Method for preparing isofagomine and its derivatives

Method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives

A novel tartaric acid salt of Isofagomine (Isofagomine tartrate) that can be used for the treatment of Gaucher disease is provided. The invention also provides a crystalline form of isofagomine tartrate, method for preparing the salt, a pharmaceutical composition containing the salt, and a method of treating Gaucher disease.

Tartrate salt of isofagomine and methods of use

The present invention provides novel hydroxy piperidine (HP) derivatives having (i) a positive charge at the position corresponding to the anomeric position of a pyranose ring; (ii) a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and (iii) a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. The linker can be absent if the lipophilic moiety corresponds to a hydrocarbon chain with a linear length of 6 or more carbons. The present invention further provides a method for treating individuals having Gaucher disease by administering the novel HP derivative as 'active-site specific chaperones' for the mutant glucocerebrosidase associated with the disease.

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Branchburg, NJ, United States of America

Kamlesh A Sheth