Prosecution Insights
Last updated: April 19, 2026
Application No. 18/473,573

Methods Of Treating Fabry Disease In Patients Having A Mutation In The GLA Gene

Non-Final OA §112
Filed
Sep 25, 2023
Examiner
NOAKES, SUZANNE MARIE
Art Unit
1656
Tech Center
1600 — Biotechnology & Organic Chemistry
Assignee
Amicus Therapeutics, Inc.
OA Round
1 (Non-Final)
73%
Grant Probability
Favorable
1-2
OA Rounds
2y 8m
To Grant
91%
With Interview

Examiner Intelligence

Grants 73% — above average
73%
Career Allow Rate
763 granted / 1047 resolved
+12.9% vs TC avg
Strong +18% interview lift
Without
With
+18.4%
Interview Lift
resolved cases with interview
Typical timeline
2y 8m
Avg Prosecution
49 currently pending
Career history
1096
Total Applications
across all art units

Statute-Specific Performance

§101
5.6%
-34.4% vs TC avg
§103
22.8%
-17.2% vs TC avg
§102
24.2%
-15.8% vs TC avg
§112
29.5%
-10.5% vs TC avg
Black line = Tech Center average estimate • Based on career data from 1047 resolved cases

Office Action

§112
DETAILED ACTION Notice of AIA Status The present application, filed on or after March 16, 2013, is being examined under the first inventor to file provisions of the AIA. Status of Application Claims 251-255 are pending and subject to examination on the merits. Priority The instant application is a DIV of US application 16987884 (now US Patent 11833164) which claims benefit of US provisional applications 62986297 and 62883759, filed 06 March 2020 and 07 August 2019, respectively . Claim Rejections - 35 USC § 112(b) The following is a quotation of 35 U.S.C. 112(b): (b ) CONCLUSION.— The specification shall conclude with one or more claims particularly pointing out and distinctly claiming the subject matter which the inventor or a joint inventor regards as the invention. Claim s 251-255 rejected under 35 U.S.C. 112(b) as being indefinite for failing to particularly point out and distinctly claim the subject matter which the inventor or a joint inventor regards as the invention. The claims recite an alpha-galactosidase A protein having specific mutations selected from the group consisting of Y184S, N228H and T412I. However, it is unclear which specific alpha-galactosidase A these amino acid positions of Y184, N228 and T412 are directed. It is well known that many alpha- galactosidase A exist across many prokaryotic and eukaryotic species such as S. cerevisiae , A. niger , Homo sapiens , etc. Furthermore, there could be several deletion or insertion variants even if the alpha-galactosidase A was directed to a specific species. It is thus suggested that the alpha-galactosidase A be directed to a specific sequence such as SEQ ID NO: 2, which is the human alpha-galactosidase A to which all mutations/substitutions in the specification correspond. In addition, claim 251 recites a list of alternatives, however, a Markush group is supposed to be a closed group of alternatives. The use of “or” between N228H and T412I makes it an open group. Simply substituting “and” for “or” will overcome this aspect of the rejection. In addition, regarding claim 255, it is noted further, again recognizing there are numerous naturally occurring alpha-galactosidase A proteins, that any increased stability should be in a direct comparison with a naturally occurring alpha-galactosidase A from the same species, having the same mutation. Claim Rejections - 35 USC § 112(a) The following is a quotation of the first paragraph of 35 U.S.C. 112(a): (a) IN GENERAL.—The specification shall contain a written description of the invention, and of the manner and process of making and using it, in such full, clear, concise, and exact terms as to enable any person skilled in the art to which it pertains, or with which it is most nearly connected, to make and use the same, and shall set forth the best mode contemplated by the inventor or joint inventor of carrying out the invention. Claims 251-255 are rejected under 35 U.S.C. 112(a) as failing to comply with the written description requirement. The claim(s) contains subject matter which was not described in the specification in such a way as to reasonably convey to one skilled in the relevant art that the inventor or a joint inventor the inventor(s), at the time the application was filed, had possession of the claimed invention. The claims are drawn to migalastat ( Galafold ® / AT1001/1-deoxygalactonojirimycin) bound to an alpha-galactosidase A protein, wherein said alpha-galactosidase A comprises a mutation selected from Y184, N228 and T412. Thus, the claims are drawn to migalastat bound to any alpha-galactosidase A protein from any species having the requisite substitutions. In addition, the claim is open and comprising and thus the mutations/substitutions of the alpha-galactosidase A protein are not only limited to those recited in the claim. Rather, said alpha-galactosidase A can any an unlimited number of additional substitutions/mutations. Thus, the genus of alpha-galactosidase A mutant enzymes, having a mutation of an amino acid of Y184, N228 or T412 in addition to/unlimited in any other mutations is enormous. The specification, admittedly discloses numerous single or double mutations of human wild-type alpha-galactosidase A comprising SEQ ID NO: 2, which have sin g le or double mutations which are identified as amendable or non-amenable substitutions (e.g. which mutations will be amenable to being helped by the binding of migalastat or not) . However, the specification is (a) drawn solely to mutations/substitutions in human alpha-galactosidase A comprising SEQ ID NO: 2, wherein not a single other alpha-galactosidase A protein from any other species is contemplated or disclosed; and (a) each of the substitutions of the human alpha-galactosidase A comprising SEQ ID NO: 2 are single, at most double, substitutions. Not an unlimited number of substitutions/mutations as currently claimed. Thus, there both are not representative of the alpha-galactosidase A proteins having substitutions/mutations, minimally of amino acids Y184, N228 or T412. As a suggestion to overcome these issues the claim could be amended to something like: A molecule comprising migalastat bound to a human alpha- galactosidase A protein , wherein said human alpha-galactosidase A protein possesses a mutation selected from the group consisting of Y184 S , N228 H and T412 I, and wherein said amino acid numbering corresponds to SEQ ID NO: 2. Reference of Interest – Not Relied Upon Prescribing information for Galafold ® ( migalastat), Amicus Therapeutics, March 2025, describing the dosing/administration recommendations and patient population having amenable alpha-galactosidase A mutations (Section 12), resulting in patients with Fabry disease responding to said migalastat therapy. While the initial approval for Galafold ® was in 2018, the amenable mutations in Section 12 was not added until 2025. Conclusion No claim is allowed. Any inquiry concerning this communication or earlier communications from the examiner should be directed to FILLIN "Examiner name" \* MERGEFORMAT SUZANNE M NOAKES whose telephone number is FILLIN "Phone number" \* MERGEFORMAT (571)272-2924 . The examiner can normally be reached FILLIN "Work Schedule?" \* MERGEFORMAT M-F (7-4) . Examiner interviews are available via telephone, in-person, and video conferencing using a USPTO supplied web-based collaboration tool. To schedule an interview, applicant is encouraged to use the USPTO Automated Interview Request (AIR) at http://www.uspto.gov/interviewpractice. If attempts to reach the examiner by telephone are unsuccessful, the examiner’s supervisor, FILLIN "SPE Name?" \* MERGEFORMAT Manjunath Rao can be reached at FILLIN "SPE Phone?" \* MERGEFORMAT 571-272-0939 . The fax phone number for the organization where this application or proceeding is assigned is 571-273-8300. Information regarding the status of published or unpublished applications may be obtained from Patent Center. Unpublished application information in Patent Center is available to registered users. To file and manage patent submissions in Patent Center, visit: https://patentcenter.uspto.gov. Visit https://www.uspto.gov/patents/apply/patent-center for more information about Patent Center and https://www.uspto.gov/patents/docx for information about filing in DOCX format. For additional questions, contact the Electronic Business Center (EBC) at 866-217-9197 (toll-free). If you would like assistance from a USPTO Customer Service Representative, call 800-786-9199 (IN USA OR CANADA) or 571-272-1000. /SUZANNE M NOAKES/ Primary Examiner, Art Unit 1656 24 February 2026
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Prosecution Timeline

Sep 25, 2023
Application Filed
Feb 24, 2026
Non-Final Rejection — §112 (current)

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Study what changed to get past this examiner. Based on 5 most recent grants.

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Prosecution Projections

1-2
Expected OA Rounds
73%
Grant Probability
91%
With Interview (+18.4%)
2y 8m
Median Time to Grant
Low
PTA Risk
Based on 1047 resolved cases by this examiner. Grant probability derived from career allow rate.

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