Rare disease drugs
These companies develop and sell drugs for rare conditions, mostly inherited ones, as their main business.
−4.6%
over 1 year · S&P 500 +11.6%
Key indicators
Health
50 / 100
Fragile
Solid
Price
P/E 23
Cheap
Expensive
Up over 1 year
33 of 63
Wallets over 90 days
1 / 2 buys / sells
Buys
Sells
Health and price: against the other tags.
Key indicators
When a figure has a bar, the marker places it among its peers, and the further right it sits, the more the figure beats the others. Green marks the best third, red the weakest and white anything in between. Some figures stay white wherever they sit, like price, pace or holding time, because neither side is better there.
The 65 companies
VRTX
+24.0%
UTHR
+28.1%
ALNY
−63.5%
Others
Size: weight in the tag. Color: move over the period.
See all companies
See all companies
V
Vertex Pharmaceuticals
VRTX
37.7%
+24.0%
I
Insmed
INSM
7.3%
−37.7%
U
United Therapeutics
UTHR
7.0%
+28.1%
A
Alnylam Pharmaceuticals
ALNY
6.1%
−63.5%
N
Neurocrine Biosciences
NBIX
4.8%
+14.0%
B
BridgeBio Pharma
BBIO
4.3%
+18.2%
B
BioMarin Pharmaceutical
BMRN
3.1%
+3.5%
K
Krystal Biotech
KRYS
2.5%
+50.3%
R
Rhythm Pharmaceuticals
RYTM
2.2%
+17.5%
M
Mirum Pharmaceuticals
MIRM
1.7%
+32.8%
S
Scholar Rock Holding
SRRK
1.5%
+40.2%
P
PTC Therapeutics
PTCT
1.5%
−15.3%
C
Cogent Biosciences
COGT
1.4%
−19.0%
L
Liquidia
LQDA
1.4%
+102.8%
I
Ionis Pharmaceuticals
IONS
1.3%
−60.0%
T
Travere Therapeutics
TVTX
1.3%
+38.9%
A
Amylyx Pharmaceuticals
AMLX
1.1%
+145.7%
I
Disc Medicine
IRON
1.1%
−7.0%
D
Dyne Therapeutics
DYN
0.9%
−7.8%
A
ACADIA Pharmaceuticals
ACAD
0.9%
−22.3%
D
Denali Therapeutics
DNLI
0.9%
+7.0%
P
Palvella Therapeutics
PVLA
0.7%
+87.0%
B
Beam Therapeutics
BEAM
0.7%
+4.4%
M
Maze Therapeutics
MAZE
0.6%
+5.4%
S
Sarepta Therapeutics
SRPT
0.5%
−10.7%
X
Xeris Biopharma
XERS
0.5%
+119.5%
B
BioCryst Pharmaceuticals
BCRX
0.5%
+6.0%
A
Agios Pharmaceuticals
AGIO
0.5%
−27.6%
E
Eton Pharmaceuticals
ETON
0.5%
+266.2%
C
Hemab Therapeutics
COAG
0.5%
S
Stoke Therapeutics
STOK
0.5%
+6.2%
P
Precigen
PGEN
0.5%
+12.9%
R
Ultragenyx Pharmaceutical
RARE
0.5%
−52.0%
T
Taysha Gene Therapies
TSHA
0.4%
−6.0%
A
Avalyn Pharma
AVLN
0.4%
S
Savara
SVRA
0.3%
+12.6%
M
MeiraGTx Holdings
MGTX
0.3%
+6.0%
I
Immix Biopharma
IMMX
0.2%
+110.8%
Z
Zevra Therapeutics
ZVRA
0.2%
−3.4%
S
Solid Biosciences
SLDB
0.2%
+44.3%
D
Design Therapeutics
DSGN
0.2%
+37.6%
C
Capricor Therapeutics
CAPR
0.2%
+62.4%
N
Neurogene
NGNE
0.1%
−23.0%
M
Monopar Therapeutics
MNPR
0.1%
−12.8%
T
Tectonic Therapeutic
TECX
0.1%
+12.1%
R
Rezolute
RZLT
0.1%
−63.5%
O
Ocugen
OCGN
<0.1%
−38.2%
F
Fulcrum Therapeutics
FULC
<0.1%
−54.9%
L
Larimar Therapeutics
LRMR
<0.1%
−10.4%
R
Rocket Pharmaceuticals
RCKT
<0.1%
−30.3%
L
Lexeo Therapeutics
LXEO
<0.1%
−68.8%
A
Arcturus Therapeutics
ARCT
<0.1%
+2.8%
T
Entrada Therapeutics
TRDA
<0.1%
−36.5%
K
Korro Bio
KRRO
<0.1%
−56.7%
P
PepGen
PEPG
<0.1%
−15.0%
S
Sionna Therapeutics
SION
<0.1%
−90.9%
I
Inovio Pharmaceuticals
INO
<0.1%
−27.0%
A
Abeona Therapeutics
ABEO
<0.1%
−51.0%
S
Spruce Biosciences
SPRB
<0.1%
−57.6%
A
Aardvark Therapeutics
AARD
<0.1%
−87.6%
A
Ascendis Pharma
ASND
+38.2%
J
Jazz Pharmaceuticals
JAZZ
+76.7%
P
Pharvaris
PHVS
+29.3%
Q
uniQure
QURE
−20.9%
S
Sol-Gel Technologies
SLGL
+75.0%