NDA/BLA filedconf 0.95

ux111 — NDA/BLA filed

$RARE · Ultragenyx Pharmaceutical Inc. · Sanfilippo syndrome Type A (MPS IIIA)

Event date

April 2, 2026

65 days ago

Sponsor runway

3 mo

$175.0M liquid

Evidence

“the U.S. Food and Drug Administration (the "FDA") has accepted for review the Company's resubmitted Biologics License Application seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome Type A (MPS IIIA).”

Source: 8-K filed 2026-04-02View on EDGAR ↗0001193125-26-139084

Comparable historical nda/bla fileds· last 5 years · same event type

Ticker	Drug	Date	Outcome
$AXSM	—	2025-12-31	unknown
$RCKT	kresladi	2025-10-14	unknown
$ALDX	reproxalap	2025-07-17	unknown
$ETON	et-600	2025-07-08	unknown
$BCRX	orladeyo (berotralstat)	2025-05-14	unknown
$CORT	relacorilant	2025-03-03	unknown
$ALDX	reproxalap	2024-11-01	unknown
$AXSM	axs-07	2024-09-04	unknown
$RCKT	kresladi	2023-10-02	unknown
$GERN	imetelstat	2023-08-21	unknown

Recent insider activity· ±90 days

15 insiders · 0 buys · 17 sells

Sell10b5-1

2026-05-18

SULIMAN SHEHNAAZ

Director

5,740 @ $25.12$144K

Grant

2026-05-14

Fust Matthew K

Director

7,751 @ ——

Grant

2026-05-14

Fust Matthew K

Director

14,058 @ ——

Grant

2026-05-14

Sanders Corazon (Corsee) D.

Director

14,058 @ ——

Grant

2026-05-14

NARACHI MICHAEL

Director

7,751 @ ——

Grant10b5-1

2026-05-14

SULIMAN SHEHNAAZ

Director

7,751 @ ——

Grant

2026-05-14

WELCH DANIEL G

Director

7,751 @ ——

Grant

2026-05-14

Ray Amrit

Director

7,751 @ ——

Grant

2026-05-14

Ray Amrit

Director

14,058 @ ——

Grant

2026-05-14

Sanders Corazon (Corsee) D.

Director

7,751 @ ——

Grant10b5-1

2026-05-14

SULIMAN SHEHNAAZ

Director

14,058 @ ——

Grant

2026-05-14

NARACHI MICHAEL

Director

14,058 @ ——

Grant

2026-05-14

Dunsire Deborah

Director

7,751 @ ——

Grant

2026-05-14

Dunsire Deborah

Director

14,058 @ ——

Grant

2026-05-14

WELCH DANIEL G

Director

14,058 @ ——

Sell

2026-05-05

Crombez Eric

EVP and Chief Medical Officer

344 @ $24.96$9K

Sell10b5-1

2026-05-01

Horn Howard

Chief Financial Officer

4,683 @ $24.90$117K

Grant

2026-04-22

Huizenga Theodore Alan

SVP, Chief Accounting Officer

20,241 @ ——

Grant

2026-04-22

Huizenga Theodore Alan

SVP, Chief Accounting Officer

7,348 @ ——

Grant

2026-04-16

Horn Howard

Chief Financial Officer

59,952 @ ——

Grant

2026-04-16

Harris Erik

EVP & Chief Commercial Officer

28,895 @ ——

Grant

2026-04-16

Harris Erik

EVP & Chief Commercial Officer

49,960 @ ——

Grant

2026-04-16

KAKKIS EMIL D

President & CEO

76,745 @ ——

Grant

2026-04-16

KAKKIS EMIL D

President & CEO

132,693 @ ——

Grant

2026-04-16

Huang Dennis Karl

See Remarks

23,116 @ ——

Grant

2026-04-16

Huang Dennis Karl

See Remarks

79,936 @ ——

Grant

2026-04-16

Pinion John Richard

See Remarks

23,116 @ ——

Grant

2026-04-16

Pinion John Richard

See Remarks

79,936 @ ——

Grant

2026-04-16

Horn Howard

Chief Financial Officer

34,674 @ ——

Grant

2026-04-16

Parschauer Karah Herdman

EVP and Chief Legal Officer

28,895 @ ——

Grant

2026-04-16

Parschauer Karah Herdman

EVP and Chief Legal Officer

49,960 @ ——

Grant

2026-04-16

Crombez Eric

EVP and Chief Medical Officer

34,674 @ ——

Grant

2026-04-16

Crombez Eric

EVP and Chief Medical Officer

59,952 @ ——

Sell10b5-1

2026-04-01

Horn Howard

Chief Financial Officer

4,683 @ $20.97$98K

Sell

2026-03-10

Parschauer Karah Herdman

EVP and Chief Legal Officer

3,508 @ $22.67$80K

Sell

2026-03-06

Parschauer Karah Herdman

EVP and Chief Legal Officer

6,018 @ $21.40$129K

Sell10b5-1

2026-03-03

Horn Howard

Chief Financial Officer

1,635 @ $22.40$37K

Sell10b5-1

2026-03-02

Horn Howard

Chief Financial Officer

3,119 @ $22.54$70K

Sell

2026-03-02

Pinion John Richard

See Remarks

10,029 @ $22.80$229K

Tax

2026-03-02

Huang Dennis Karl

See Remarks

999 @ $23.39$23K

Sell

2026-03-02

KAKKIS EMIL D

President & CEO

54,404 @ $22.80$1.24M

Tax

2026-03-02

Huizenga Theodore Alan

SVP, Chief Accounting Officer

148 @ $23.39$3K

Sell

2026-03-02

Huizenga Theodore Alan

SVP, Chief Accounting Officer

1,632 @ $22.80$37K

Sell

2026-03-02

Crombez Eric

EVP and Chief Medical Officer

7,029 @ $22.80$160K

Sell

2026-03-02

Parschauer Karah Herdman

EVP and Chief Legal Officer

8,135 @ $22.80$185K

Sell

2026-03-02

Harris Erik

EVP & Chief Commercial Officer

10,539 @ $22.80$240K

Sell10b5-1

2026-03-02

Horn Howard

Chief Financial Officer

5,290 @ $22.80$121K

Grant

2026-03-01

Crombez Eric

EVP and Chief Medical Officer

11,643 @ ——

Grant

2026-03-01

Pinion John Richard

See Remarks

11,137 @ ——

Grant

2026-03-01

KAKKIS EMIL D

President & CEO

71,177 @ ——

+ 6 more

Date	Insider	Action	Shares	Price	Value
2026-05-18	SULIMAN SHEHNAAZDirector	Sell10b5-1	5,740	$25.12	$144K
2026-05-14	Fust Matthew KDirector	Grant	7,751	—	—
2026-05-14	Fust Matthew KDirector	Grant	14,058	—	—
2026-05-14	Sanders Corazon (Corsee) D.Director	Grant	14,058	—	—
2026-05-14	NARACHI MICHAELDirector	Grant	7,751	—	—
2026-05-14	SULIMAN SHEHNAAZDirector	Grant10b5-1	7,751	—	—
2026-05-14	WELCH DANIEL GDirector	Grant	7,751	—	—
2026-05-14	Ray AmritDirector	Grant	7,751	—	—
2026-05-14	Ray AmritDirector	Grant	14,058	—	—
2026-05-14	Sanders Corazon (Corsee) D.Director	Grant	7,751	—	—
2026-05-14	SULIMAN SHEHNAAZDirector	Grant10b5-1	14,058	—	—
2026-05-14	NARACHI MICHAELDirector	Grant	14,058	—	—
2026-05-14	Dunsire DeborahDirector	Grant	7,751	—	—
2026-05-14	Dunsire DeborahDirector	Grant	14,058	—	—
2026-05-14	WELCH DANIEL GDirector	Grant	14,058	—	—
2026-05-05	Crombez EricEVP and Chief Medical Officer	Sell	344	$24.96	$9K
2026-05-01	Horn HowardChief Financial Officer	Sell10b5-1	4,683	$24.90	$117K
2026-04-22	Huizenga Theodore AlanSVP, Chief Accounting Officer	Grant	20,241	—	—
2026-04-22	Huizenga Theodore AlanSVP, Chief Accounting Officer	Grant	7,348	—	—
2026-04-16	Horn HowardChief Financial Officer	Grant	59,952	—	—
2026-04-16	Harris ErikEVP & Chief Commercial Officer	Grant	28,895	—	—
2026-04-16	Harris ErikEVP & Chief Commercial Officer	Grant	49,960	—	—
2026-04-16	KAKKIS EMIL DPresident & CEO	Grant	76,745	—	—
2026-04-16	KAKKIS EMIL DPresident & CEO	Grant	132,693	—	—
2026-04-16	Huang Dennis KarlSee Remarks	Grant	23,116	—	—
2026-04-16	Huang Dennis KarlSee Remarks	Grant	79,936	—	—
2026-04-16	Pinion John RichardSee Remarks	Grant	23,116	—	—
2026-04-16	Pinion John RichardSee Remarks	Grant	79,936	—	—
2026-04-16	Horn HowardChief Financial Officer	Grant	34,674	—	—
2026-04-16	Parschauer Karah HerdmanEVP and Chief Legal Officer	Grant	28,895	—	—
2026-04-16	Parschauer Karah HerdmanEVP and Chief Legal Officer	Grant	49,960	—	—
2026-04-16	Crombez EricEVP and Chief Medical Officer	Grant	34,674	—	—
2026-04-16	Crombez EricEVP and Chief Medical Officer	Grant	59,952	—	—
2026-04-01	Horn HowardChief Financial Officer	Sell10b5-1	4,683	$20.97	$98K
2026-03-10	Parschauer Karah HerdmanEVP and Chief Legal Officer	Sell	3,508	$22.67	$80K
2026-03-06	Parschauer Karah HerdmanEVP and Chief Legal Officer	Sell	6,018	$21.40	$129K
2026-03-03	Horn HowardChief Financial Officer	Sell10b5-1	1,635	$22.40	$37K
2026-03-02	Horn HowardChief Financial Officer	Sell10b5-1	3,119	$22.54	$70K
2026-03-02	Pinion John RichardSee Remarks	Sell	10,029	$22.80	$229K
2026-03-02	Huang Dennis KarlSee Remarks	Tax	999	$23.39	$23K
2026-03-02	KAKKIS EMIL DPresident & CEO	Sell	54,404	$22.80	$1.24M
2026-03-02	Huizenga Theodore AlanSVP, Chief Accounting Officer	Tax	148	$23.39	$3K
2026-03-02	Huizenga Theodore AlanSVP, Chief Accounting Officer	Sell	1,632	$22.80	$37K
2026-03-02	Crombez EricEVP and Chief Medical Officer	Sell	7,029	$22.80	$160K
2026-03-02	Parschauer Karah HerdmanEVP and Chief Legal Officer	Sell	8,135	$22.80	$185K
2026-03-02	Harris ErikEVP & Chief Commercial Officer	Sell	10,539	$22.80	$240K
2026-03-02	Horn HowardChief Financial Officer	Sell10b5-1	5,290	$22.80	$121K
2026-03-01	Crombez EricEVP and Chief Medical Officer	Grant	11,643	—	—
2026-03-01	Pinion John RichardSee Remarks	Grant	11,137	—	—
2026-03-01	KAKKIS EMIL DPresident & CEO	Grant	71,177	—	—

+ 6 more transactions

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