Market Cap 2.32B
Revenue (ttm) 560.23M
Net Income (ttm) -569.18M
EPS (ttm) N/A
PE Ratio 0.00
Forward PE N/A
Profit Margin -101.60%
Debt to Equity Ratio 0.00
Volume 679,216
Avg Vol 2,735,760
Day's Range N/A - N/A
Shares Out 96.48M
Stochastic %K 67%
Beta 0.15
Analysts Strong Sell
Price Target $67.40

Company Profile

Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in North America, Latin America, Europe, the Middle East, Africa, and the Asia-Pacific. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevi...

Industry: Biotechnology
Sector: Healthcare
Phone: 415 483 8800
Address:
60 Leveroni Court, Novato, United States
LegacyLiquidity
LegacyLiquidity Feb. 4 at 9:51 AM
$RARE biotech, gene therapy leader
0 · Reply
TymeTryethTroth
TymeTryethTroth Feb. 3 at 2:26 PM
$XERS https://seekingalpha.com/article/4865261-xeris-biopharma-strong-recorlev-momentum-and-pipeline-opportunity?share_source=shared_news $BMRN $RARE $LLY $CORT (Comps mentioned in article)
0 · Reply
ZacksResearch
ZacksResearch Feb. 2 at 4:15 PM
FDA round two for $RARE — this one isn’t over yet ⚠️ Ultragenyx resubmitted the UX111 BLA for Sanfilippo syndrome after a CRL, this time adding long-term neurologic and biomarker data. The FDA decision is now targeted for Q3 2026, putting a clear regulatory timeline back on the table. What does this resubmission really change for RARE? Full details here 👉 https://www.zacks.com/stock/news/2827196/ultragenyx-pharmaceutical-resubmits-ux111-bla-for-sanfilippo-syndrome?cid=sm-stocktwits-2-2827196-teaser-31462&ADID=SYND_STOCKTWITS_TWEET_2_2827196_TEASER_31462
0 · Reply
ZacksResearch
ZacksResearch Feb. 2 at 3:15 PM
$RARE resubmits BLA for UX111, addressing FDA's CMC concerns with additional data and extended clinical follow-up for MPS IIIA treatment. Shares have fallen 44.6% over the past year. Discover the potential impact on RARE here 👉 https://www.zacks.com/stock/news/2827196/ultragenyx-pharmaceutical-resubmits-ux111-bla-for-sanfilippo-syndrome?cid=sm-stocktwits-2-2827196-body-31463&ADID=SYND_STOCKTWITS_TWEET_2_2827196_BODY_31463
0 · Reply
Masonsy
Masonsy Jan. 31 at 1:09 AM
$RARE Next week, Ultragenyx will present the robust latest UX111 clinical data at WORLDSymposium 2026. One more week later, the company will release Q4 2025 earnings, 2026 guidance, and cost reduction details. Stay tuned.
0 · Reply
StocktwitsNews
StocktwitsNews Jan. 30 at 4:51 PM
RARE Stock Slips 3% Today — What’s Up With Ultragenyx’s Gene Therapy For Sanfilippo Syndrome? $RARE https://stocktwits.com/news/equity/markets/rare-stock-slips-after-resubmitting-application-for-gene-therapy/cmyS8PXR4Si
0 · Reply
TwongStocks
TwongStocks Jan. 30 at 1:31 PM
$RARE Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA https://www.globenewswire.com/news-release/2026/01/30/3229517/20739/en/Ultragenyx-Resubmits-Biologics-License-Application-for-UX111-AAV-Gene-Therapy-to-Treat-Sanfilippo-Syndrome-Type-A-MPS-IIIA-to-U-S-FDA.html Company expects up to six-month review period per FDA guidelines
0 · Reply
JFais
JFais Jan. 28 at 9:34 PM
Surprised $RARE not down more re two gene therapy regulatory decisions this year (FDA unpredictability)
0 · Reply
GunnarMeister
GunnarMeister Jan. 23 at 1:07 PM
$RARE the short term next catalyst is when they will announce layoffs and layoff’s seemingly around early Feb. afterwards, need flawless execution of their 2 gene therapies.
0 · Reply
JoeyStocks26
JoeyStocks26 Jan. 21 at 6:39 PM
$RARE bottom ?
1 · Reply
Latest News on RARE
Ultragenyx's bone disease drug fails late-stage trials

Dec 29, 2025, 8:35 AM EST - 5 weeks ago

Ultragenyx's bone disease drug fails late-stage trials


Ultragenyx to Participate at Investor Conferences in September

Aug 29, 2025, 4:30 PM EDT - 5 months ago

Ultragenyx to Participate at Investor Conferences in September


LegacyLiquidity
LegacyLiquidity Feb. 4 at 9:51 AM
$RARE biotech, gene therapy leader
0 · Reply
TymeTryethTroth
TymeTryethTroth Feb. 3 at 2:26 PM
$XERS https://seekingalpha.com/article/4865261-xeris-biopharma-strong-recorlev-momentum-and-pipeline-opportunity?share_source=shared_news $BMRN $RARE $LLY $CORT (Comps mentioned in article)
0 · Reply
ZacksResearch
ZacksResearch Feb. 2 at 4:15 PM
FDA round two for $RARE — this one isn’t over yet ⚠️ Ultragenyx resubmitted the UX111 BLA for Sanfilippo syndrome after a CRL, this time adding long-term neurologic and biomarker data. The FDA decision is now targeted for Q3 2026, putting a clear regulatory timeline back on the table. What does this resubmission really change for RARE? Full details here 👉 https://www.zacks.com/stock/news/2827196/ultragenyx-pharmaceutical-resubmits-ux111-bla-for-sanfilippo-syndrome?cid=sm-stocktwits-2-2827196-teaser-31462&ADID=SYND_STOCKTWITS_TWEET_2_2827196_TEASER_31462
0 · Reply
ZacksResearch
ZacksResearch Feb. 2 at 3:15 PM
$RARE resubmits BLA for UX111, addressing FDA's CMC concerns with additional data and extended clinical follow-up for MPS IIIA treatment. Shares have fallen 44.6% over the past year. Discover the potential impact on RARE here 👉 https://www.zacks.com/stock/news/2827196/ultragenyx-pharmaceutical-resubmits-ux111-bla-for-sanfilippo-syndrome?cid=sm-stocktwits-2-2827196-body-31463&ADID=SYND_STOCKTWITS_TWEET_2_2827196_BODY_31463
0 · Reply
Masonsy
Masonsy Jan. 31 at 1:09 AM
$RARE Next week, Ultragenyx will present the robust latest UX111 clinical data at WORLDSymposium 2026. One more week later, the company will release Q4 2025 earnings, 2026 guidance, and cost reduction details. Stay tuned.
0 · Reply
StocktwitsNews
StocktwitsNews Jan. 30 at 4:51 PM
RARE Stock Slips 3% Today — What’s Up With Ultragenyx’s Gene Therapy For Sanfilippo Syndrome? $RARE https://stocktwits.com/news/equity/markets/rare-stock-slips-after-resubmitting-application-for-gene-therapy/cmyS8PXR4Si
0 · Reply
TwongStocks
TwongStocks Jan. 30 at 1:31 PM
$RARE Ultragenyx Resubmits Biologics License Application for UX111 AAV Gene Therapy to Treat Sanfilippo Syndrome Type A (MPS IIIA) to U.S. FDA https://www.globenewswire.com/news-release/2026/01/30/3229517/20739/en/Ultragenyx-Resubmits-Biologics-License-Application-for-UX111-AAV-Gene-Therapy-to-Treat-Sanfilippo-Syndrome-Type-A-MPS-IIIA-to-U-S-FDA.html Company expects up to six-month review period per FDA guidelines
0 · Reply
JFais
JFais Jan. 28 at 9:34 PM
Surprised $RARE not down more re two gene therapy regulatory decisions this year (FDA unpredictability)
0 · Reply
GunnarMeister
GunnarMeister Jan. 23 at 1:07 PM
$RARE the short term next catalyst is when they will announce layoffs and layoff’s seemingly around early Feb. afterwards, need flawless execution of their 2 gene therapies.
0 · Reply
JoeyStocks26
JoeyStocks26 Jan. 21 at 6:39 PM
$RARE bottom ?
1 · Reply
BillionerOfKing
BillionerOfKing Jan. 21 at 12:13 PM
$RARE Current Stock Price: $22.64 Contracts to trade: $20 RARE Feb 20 2026 Call Entry: $3.87 Exit: $7.68 ROI: 98% Hold ~23 days Shared as daily free alerts and for educational purposes only. https://dailypickai.com/freealerts
0 · Reply
LoveRetailFroth
LoveRetailFroth Jan. 20 at 8:25 PM
0 · Reply
shortvolume
shortvolume Jan. 20 at 8:10 PM
$RARE Thirty-Five
0 · Reply
biotech
biotech Jan. 16 at 9:03 AM
$SGMO $XBI Therapies to Watch in 1H 2026 $RGNX DMD Gene Therapy RGX-202 $RARE Glycogen Storage Disease Gene Therapy DTX401 $SLDB Friedreich Ataxia Gene Therapy SGT-212 $SGMO Fabry gene therapy isaralgagene civaparvove ( ST-920) https://www.cgtlive.com/view/therapies-watch-1h-2026
0 · Reply
lidopete
lidopete Jan. 15 at 12:35 PM
$MREO $MREO $RARE GROK Generated: Why This Matters for Mereo BioPharma: "This development is a major positive indicator for MREO's regulatory package preparation." Yes, the FDA's December 2025 qualification of total hip bone mineral density (BMD) (measured by DXA) as a validated surrogate endpoint for fracture reduction in osteoporosis drug trials (specifically for post-menopausal women at risk of fracture) represents a significant regulatory shift. "This FDA move is a strong, mechanistic/regulatory tailwind for setrusumab's profile and could meaningfully de-risk future discussions with agencies for this ultra-rare indication". Announced on December 19, 2025, this allows BMD percentage change from baseline (at 24 months) to serve as a primary endpoint in Phase 3 trials, replacing the traditional (and more burdensome) requirement for fracture incidence as the primary measure.
0 · Reply
roaringcharts
roaringcharts Jan. 14 at 3:24 PM
$VXX $ROLR $RARE High Roller Technologies Partners with Crypto.com to Enter the Rapidly Growing Prediction Markets Space The binding strategic partnership agreement with Crypto.com to power event-based Prediction Markets, with a Mature Market Estimated to exceed $1.X Trillion in annual trading volume1 Las Vegas, Nevada, Jan. 14, 2026 (GLOBE NEWSWIRE) -- High Roller Technologies, Inc. (“High Roller”) (NYSE: ROLR), operator of the award-winning, premium online casino brands High Roller and Fruta, today announced it has entered into a binding Letter of Intent (the “LOI”) with Crypto.com | Derivatives North America (“CDNA”), for an exclusive partnership to launch an event-based prediction markets product in the United States of America.
0 · Reply
roaringcharts
roaringcharts Jan. 14 at 12:24 AM
$RARE Still in watch https://x.com/i/status/2010693911732187382
0 · Reply
roaringcharts
roaringcharts Jan. 13 at 2:40 PM
$RARE $EVTV $SPRC REAL COMPANY REAL REVS APPROVED DRUGS CASH ON HAND Preliminary 2025 total revenue of $672 million to $674 million, exceeding top end of guidance Preliminary cash and investments of approximately $735 million as of December 31, 2025 Anticipated 2026 catalysts include two potential approvals and pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome
0 · Reply
NoOriginality
NoOriginality Jan. 13 at 1:14 PM
$BCTX Eyes are still on $RARE for January biotech gains.
0 · Reply
roaringcharts
roaringcharts Jan. 13 at 12:43 PM
$RARE steady climb
0 · Reply
roaringcharts
roaringcharts Jan. 13 at 10:38 AM
$RARE they're buying SG Americas Securities LLC established a new position in Ultragenyx by purchasing 125,429 shares in Q3, worth about $3.77 million and representing roughly 0.13% of the company.
0 · Reply
OptionRunners
OptionRunners Jan. 12 at 7:52 PM
$RARE bull risk reversals
0 · Reply