PDUFAconf 0.85

ux111 — PDUFA

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

Event date

Date TBD

Sponsor runway

3 mo

$175.0M liquid

Evidence

“BLA resubmitted to FDA with PDUFA date expected in third quarter 2026. These longer-term data were included in the resubmitted Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for UX111. The Company anticipates up to a six-month review period from the date of resubmission per FDA regulations, with a PDUFA date expected in the third quarter of 2026.”

Source: 8-K filed 2026-02-03View on EDGAR ↗0001193125-26-034474

Comparable historical pdufas· last 5 years · same event type

75% hit rate (3/4)

Ticker	Drug	Date	Outcome
$RCKT	kresladi	2026-03-28	success
$RYTM	imcivree	2026-03-20	success
$CYTK	aficamten	2025-12-26	unknown
$ALDX	reproxalap	2025-12-16	unknown
$BCRX	orladeyo	2025-12-12	success
$OMER	narsoplimab	2025-09-25	unknown
$BCRX	orladeyo (berotralstat)	2025-09-12	unknown
$ETON	et-400	2025-05-28	unknown
$ALDX	reproxalap	2025-04-02	failure
$IONS	olezarsen	2024-12-19	unknown

Recent insider activity· ±90 days

15 insiders · 0 buys · 5 sells

Sell10b5-1

2026-05-18

SULIMAN SHEHNAAZ

Director

5,740 @ $25.12$144K

Grant

2026-05-14

Ray Amrit

Director

7,751 @ ——

Grant

2026-05-14

Dunsire Deborah

Director

7,751 @ ——

Grant10b5-1

2026-05-14

SULIMAN SHEHNAAZ

Director

7,751 @ ——

Grant10b5-1

2026-05-14

SULIMAN SHEHNAAZ

Director

14,058 @ ——

Grant

2026-05-14

WELCH DANIEL G

Director

7,751 @ ——

Grant

2026-05-14

WELCH DANIEL G

Director

14,058 @ ——

Grant

2026-05-14

Sanders Corazon (Corsee) D.

Director

7,751 @ ——

Grant

2026-05-14

Dunsire Deborah

Director

14,058 @ ——

Grant

2026-05-14

Ray Amrit

Director

14,058 @ ——

Grant

2026-05-14

Sanders Corazon (Corsee) D.

Director

14,058 @ ——

Grant

2026-05-14

Fust Matthew K

Director

14,058 @ ——

Grant

2026-05-14

NARACHI MICHAEL

Director

14,058 @ ——

Grant

2026-05-14

NARACHI MICHAEL

Director

7,751 @ ——

Grant

2026-05-14

Fust Matthew K

Director

7,751 @ ——

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

7,348 @ ——

Grant

2026-04-22

Huizenga Theodore Alan

SVP, Chief Accounting Officer

20,241 @ ——

Grant

2026-04-16

Crombez Eric

EVP and Chief Medical 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

Horn Howard

Chief Financial Officer

59,952 @ ——

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 @ ——

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

Date	Insider	Action	Shares	Price	Value
2026-05-18	SULIMAN SHEHNAAZDirector	Sell10b5-1	5,740	$25.12	$144K
2026-05-14	Ray AmritDirector	Grant	7,751	—	—
2026-05-14	Dunsire DeborahDirector	Grant	7,751	—	—
2026-05-14	SULIMAN SHEHNAAZDirector	Grant10b5-1	7,751	—	—
2026-05-14	SULIMAN SHEHNAAZDirector	Grant10b5-1	14,058	—	—
2026-05-14	WELCH DANIEL GDirector	Grant	7,751	—	—
2026-05-14	WELCH DANIEL GDirector	Grant	14,058	—	—
2026-05-14	Sanders Corazon (Corsee) D.Director	Grant	7,751	—	—
2026-05-14	Dunsire DeborahDirector	Grant	14,058	—	—
2026-05-14	Ray AmritDirector	Grant	14,058	—	—
2026-05-14	Sanders Corazon (Corsee) D.Director	Grant	14,058	—	—
2026-05-14	Fust Matthew KDirector	Grant	14,058	—	—
2026-05-14	NARACHI MICHAELDirector	Grant	14,058	—	—
2026-05-14	NARACHI MICHAELDirector	Grant	7,751	—	—
2026-05-14	Fust Matthew KDirector	Grant	7,751	—	—
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	7,348	—	—
2026-04-22	Huizenga Theodore AlanSVP, Chief Accounting Officer	Grant	20,241	—	—
2026-04-16	Crombez EricEVP and Chief Medical 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	Horn HowardChief Financial Officer	Grant	59,952	—	—
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	—	—
2026-04-01	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

← All catalysts for $RARE